Category Archives: Gene therapy

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Fred Hutch virologists Martine Aubert, PhD, and Keith Jerome, MD, PhD, are doing laboratory experiments to develop a gene therapy aimed at curing herpes

Credit: Fred Hutch Cancer Center

SEATTLE May 13, 2024 Researchers at Fred Hutch Cancer Center have found in pre-clinical studies that an experimental gene therapy for genital and oral herpes removed 90% or more of the infection and suppressed how much virus can be released from an infected individual, which suggests that the therapy would also reduce the spread of the virus.

Herpes is very sneaky. It hides out among nerve cells and then reawakens and causes painful skin blisters, said Keith Jerome, MD, PhD, professor in the Vaccine and Infectious Disease Division at Fred Hutch. Our aim is to cure people of this infection, so that they dont have to live with the worry of outbreaks or of transmitting it to another person.

Published May 13 in Nature Communications, Jerome and his Fred Hutch team published an encouraging step toward a gene therapy for herpes.

The experimental gene therapy involves injecting into the blood a mixture of gene editing molecules that seek out where the herpes virus resides in the body. The mixture includes laboratory-modified viruses called a vector commonly used in gene therapies plus enzymes that work like molecular scissors. Once the vector reaches the clusters of nerves where the herpes virus hangs out, the molecular scissors snip away at the herpes viruss genes to damage them or remove the virus entirely.

We are using a meganuclease enzyme that cuts in two different places in the herpes viruss DNA, said first author Martine Aubert, PhD, principal staff scientist at Fred Hutch. These cuts damage the virus so much that it cant repair itself. Then the bodys own repair systems recognize the damaged DNA as foreign and get rid of it.

Using mouse models of the infection, the experimental therapy eliminated 90% of herpes simplex virus 1 (HSV-1) after facial infection, also known as oral herpes, and 97% of herpes HSV-1 after genital infection. It took about a month for the treated mice to show these reductions, and the reduction of virus seemed to get more complete over time.

In addition, the researchers found that the HSV-1 gene therapy had a significant reduction in both the frequency and amount of viral shedding.

If you talk to people living with herpes, many are worried about whether their infection will transmit to others, Jerome said. Our new study shows that we can reduce both the amount of virus within the body and how much virus is shed.

The Fred Hutch team also simplified their gene editing treatment, making it safer and easier to make. In a 2020 study, they used three vectors and two different meganucleases. The latest study uses just one vector and one meganuclease capable of cutting the virus DNA in two places.

Our streamlined gene editing approach is effective at eliminating the herpes virus and has less side effects to the liver and nerves, Jerome said. This suggests that the therapy will be safer for people and easier to make, since it has fewer ingredients.

While the Fred Hutch scientists are encouraged by how well the gene therapy works in animal models and are eager to translate the findings to treatments for people, they are also careful about the steps needed to prepare for clinical trials. They also noted that though the current study examined HSV-1 infections, they are working on adapting the gene editing technology to target HSV-2 infections.

Were collaborating with numerous partners as we approach clinical trials so we align with federal regulators to ensure safety and effectiveness of the gene therapy, Jerome said. We deeply appreciate the support of herpes advocates as they share our vision for curing this infection.

Herpes simplex virus (HSV) is a common infection that lasts a lifetime once people are infected. Current therapies can only suppress but not completely eliminate symptoms, which include painful blisters. According to the World Health Organization, an estimated 3.7 billion people under the age of 50 (67%) have HSV-1, which causes oral herpes. An estimated 491 million people aged 15-49 (13%) worldwide have HSV-2, which causes genital herpes.

Herpes can create other harms to peoples health. HSV-2 increases the risk of acquiring HIV infection. Other studies have linked dementia with HSV-1.

The work was funded by the National Institutes of Health, the Caladan Foundation and more than 2,000 donors. The meganucleases used in this research are derivatives of commercially-available meganucleases.

Note: Scientists at Fred Hutch played a role in developing these discoveries, and Fred Hutch and certain of its scientists may benefit financially from this work in the future.

###

Media contact: Molly McElroy mwmcelro@fredhutch.org

Fred Hutchinson Cancer Center unites individualized care and advanced research to provide the latest cancer treatment options while accelerating discoveries that prevent, treat and cure cancer and infectious diseases worldwide.

Based in Seattle, Fred Hutch is an independent, nonprofit organization and the only National Cancer Institute-designated cancer center in Washington. We have earned a global reputation for our track record of discoveries in cancer, infectious disease and basic research, including important advances in bone marrow transplantation, immunotherapy, HIV/AIDS prevention and COVID-19 vaccines. Fred Hutch operates eight clinical care sites that provide medical oncology, infusion, radiation, proton therapy and related services. Fred Hutch also serves as UW Medicines cancer program.

Nature Communications

Experimental study

Animals

Gene editing for latent herpes simplex virus infection reduces viral load and shedding in vivo

13-May-2024

Disclaimer: AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert system.

Read more here:
Herpes cure with gene editing makes progress in laboratory studies - EurekAlert

The experimental therapy eliminated 90 percent of HSV-1 after facial infection and 97 percent of HSV-1 after genital infection.

Scientists at the Fred Hutch Cancer Center have discovered that an experimental gene therapy for genital and oral herpes removed 90 percent or more of the infection and suppressed how much virus can be released from an infected individual, suggesting that the therapy would also lessen the transmission of the virus.

Herpes simplex virus (HSV) is a common infection that lasts a lifetime once people are infected. According to the World Health Organization, an estimated 3.7 billion people under the age of 50 have HSV-1, which causes oral herpes and has been linked to dementia. An estimated 491 million people aged 15-49 globally have HSV-2, which causes genital herpes and increases the risk of acquiring HIV. At present, HSV therapies only suppress symptoms.

Dr Keith Jerome, professor in the Vaccine and Infectious Disease Division at Fred Hutch, stated: Herpes is very sneaky. It hides out among nerve cells and then reawakens and causes painful skin blistersOur aim is to cure people of this infection, so that they dont have to live with the worry of outbreaks or of transmitting it to another person.

The experimental gene therapy involves injecting a mixture of gene editing molecules into the blood that find where the herpes virus resides in the body. The mixture includes a vector, as well as enzymes. When the vector reaches the clusters of nerves where the herpes virus resides, the enzymes cut the herpes viruss genes to damage them or remove the virus completely.

First author DrMartine Aubert, principal staff scientist at Fred Hutch, explained: We are using a meganuclease enzyme that cuts in two different places in the herpes viruss DNAThese cuts damage the virus so much that it cant repair itself. Then the bodys own repair systems recognise the damaged DNA as foreign and get rid of it.

In mouse models of the infection, the experimental therapy eliminated 90 percent of HSV-1 after facial infection and 97 percent of HSV-1 after genital infection. It took around a month for the treated mice to show these reductions, and over time, the reduction of virus appeared to become more complete.

Additionally, the team saw that the HSV-1 gene therapy had a significant reduction in both the frequency and amount of viral shedding. If you talk to people living with herpes, many are worried about whether their infection will transmit to others, Dr Jerome commented. Our new study shows that we can reduce both the amount of virus within the body and how much virus is shed.

Furthermore, the teams gene editing treatment is safer and easier to manufacture, using just one vector and one meganuclease. Dr Jerome said: Our streamlined gene editing approach is effective at eliminating the herpes virus and has less side effects to the liver and nervesThis suggests that the therapy will be safer for people and easier to make, since it has fewer ingredients.

Although the team is hopeful about the gene therapy, considering its efficacy in animal models, they are careful about the steps required to prepare for clinical trials. Were collaborating with numerous partners as we approach clinical trials so we align with federal regulators to ensure safety and effectiveness of the gene therapy, Dr Jerome concluded.

The team noted that they are working on adapting the gene editing technology to target HSV-2 infections as well.

This study was published inNature Communications.

Read the rest here:
Gene editing for HSV-1 shows significant reduction of viral load - Drug Target Review

Marketresearch.biz reports that the The Global Retrovirus-Based Gene Therapy Drugs Market size is expected to be worth around USD 14.2 Billion by 2033, from USD 2.2 Billion in 2023, growing at a CAGR of 21.1% during the forecast period from 2024 to 2033.

The Retrovirus-Based Gene Therapy Drugs Market report serves as a comprehensive resource, synthesizing data from various reputable sources such as government bodies, industry associations, and established companies. This rich data repository not only validates the research findings but also assists clients in making informed decisions. By offering a contemporary understanding of the Retrovirus-Based Gene Therapy Drugs Market dynamics, the report equips stakeholders with actionable insights.

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The Retrovirus-Based Gene Therapy Drugs market is experiencing rapid growth due to advancements in genetic engineering and promising results in clinical trials. This market segment utilizes retroviruses as vectors to deliver therapeutic genes into patients cells, treating various genetic disorders and cancers. The market is driven by increasing investment in biotechnology research, rising prevalence of genetic diseases, and regulatory approvals for new gene therapy drugs. However, high treatment costs and potential safety concerns remain key challenges for market expansion.

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The Retrovirus-Based Gene Therapy Drugs market is characterized by competitive dynamics and a growing number of players entering the field. Established biotech companies and academic institutions are actively involved in research and development to enhance treatment efficacy and safety. The market is not yet saturated, presenting opportunities for new entrants and innovations in vector technology and therapeutic applications. As more clinical data becomes available and regulatory frameworks evolve, the market is poised for continued growth and expansion in treating a wide range of genetic disorders and cancers.

By Type

By Target Disease

By Delivery Method

By End Users

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Retrovirus-Based Gene Therapy Drugs Market to Reach USD 14.2 Billion by 2033, Growing at 21.1% CAGR - PharmiWeb.com

We are in an unprecedented time in neurotherapeutics. Medicines that address the causative disease biology underlying central nervous system (CNS) diseases such as Alzheimers disease and amyotrophic lateral sclerosis (ALS) are becoming a reality for patients.

Despite these advancements, progress is limited by delivery challenges, as the blood-brain barrier prevents the uptake of many investigational therapies. At Voyager Therapeutics, we are committed to overcoming this challenge and delivering transformative neurogenetic medicines to patients in need. We are approaching this with our TRACER (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) capsid discovery platform. Voyager has leveraged TRACER to create novel capsids that harness the extensive vasculature of the CNS to cross the blood-brain barrier, with the potential to enable gene therapies for CNS diseases.

TRACER-generated capsids have been shown in preclinical studies to transduce a broad range of CNS regions and cell types following intravenous delivery, while simultaneously de-targeting the liver, an organ often implicated in side effects. In cross-species preclinical studies (rodents and multiple non-human primate species), intravenous delivery of TRACER-generated capsids resulted in widespread payload expression across the CNS at relatively low doses. These studies enabled selection of multiple development candidates in Voyagers wholly-owned and partnered gene therapy programs for neurologic diseases, which are now advancing towards human clinical trials.

The strong performance of our capsids has enabled selection of three development candidates in Voyagers wholly-owned and partnered gene therapy programs for neurologic diseases, which are currently advancing towards anticipated investigational new drug (IND) application filings in 2025.

Voyagers team presented data for our second-generation, TRACER-generated capsids and CNS gene therapy programs advancing toward clinical trials.

Through our TRACER platform, we are reaching CNS targets once thought unreachable. Key findings presented at this years ASGCT annual meeting across species, capsid generations, and disease models provided the most extensive validation to date of the high translational potential of our TRACER capsids for gene therapy in the CNS:

Voyagers novel TRACER-derived capsids underlie 13 partnered programs and three wholly-owned programs to enable IV-delivery of gene therapies for diseases of the central nervous system. We are proud of the progress of our programs, propelled by our vision to help create a world in which transformative treatments and cures are available to the millions afflicted with neurological diseases.

See the rest here:
Our Mission at Voyager Therapeutics: Define the Future of Neurogenetic Medicines - BioSpace

WEDNESDAY, May 15, 2024 (HealthDay News) -- An experimental gene therapy could one day provide a first-ever cure for genital and oral herpes, researchers report.

The gene therapy removed 90% or more of oral herpes infection in lab mice, and it also suppressed how much virus an infected animal shed, according to results published May 13 in the journal Nature Communications.

The experimental therapy involves an injection of gene-editing molecules that seek out herpes virus hiding in the body, researchers said.

Herpes is very sneaky. It hides out among nerve cells and then reawakens and causes painful skin blisters, explained researcher Dr. Keith Jerome, a professor in the Vaccine and Infectious Disease Division at Fred Hutch Cancer Center in Seattle.

The gene-editing cocktail includes laboratory-modified delivery viruses that contain an enzyme that works like molecular scissors, the researchers said.

Once the delivery virus reaches a cluster of nerves where herpes is hiding, it releases the enzyme. The enzyme then snips away at the herpes virus's genes, to either damage or destroy it.

The enzyme cuts in two different places in the herpes viruss DNA, said lead researcher Martine Albert, a principal staff scientist at Fred Hutch. These cuts damage the virus so much that it cant repair itself. Then the bodys own repair systems recognize the damaged DNA as foreign and get rid of it.

An estimated 3.7 billion people younger than 50 have herpes simplex virus 1, which causes oral herpes, according to the World Health Organization.

Another 491 million people 15 to 49 have herpes simplex 2, which causes genital herpes.

This experimental therapy eliminated 90% of facial infection and 97% of genital infection in lab mice who were infected with herpes simplex 1, researchers say.

It took about a month for the treated mice to reach these reductions, and the reduction of virus appeared to become more complete over time.

If you talk to people living with herpes, many are worried about whether their infection will transmit to others, Jerome said in a cancer center news release. Our new study shows that we can reduce both the amount of virus within the body and how much virus is shed.

This new therapy represents a streamlined approach to attacking herpes.

In a 2020 study, the research team used three different delivery viruses armed with two different enzymes to attack herpes. This study used just one delivery virus and one enzyme capable of cutting the virus DNA in two places.

Our streamlined gene-editing approach is effective at eliminating the herpes virus and has less side effects to the liver and nerves, Jerome said. This suggests that the therapy will be safer for people and easier to make, since it has fewer ingredients.

Researchers are preparing to translate the findings into treatments for humans that can be tested in clinical trials.

Were collaborating with numerous partners as we approach clinical trials so we align with federal regulators to ensure safety and effectiveness of the gene therapy, Jerome said.

The team also is adapting the gene-editing technology to target herpes simplex 2 viruses.

More information

The Cleveland Clinic has more about herpes simplex.

SOURCE: Fred Hutch Cancer Center, news release, May 13, 2024

Excerpt from:
Early Hints at a Gene Therapy Cure for Herpes - HealthDay

Findings highlight the advantages of Adolore's approach for the delivery of proprietary gene therapy directly to specialized pain-sensing peripheral nerves (nociceptors) that mediate profound analgesia with the potential to address the great unmet need for non-opioid chronic pain therapies

Data further supports the clinical-translational value of Adolore's proprietary non-opioid analgesics for treating chronic non-cancer pain

Company continuing progress with IND-enabling studies of ADLR-1001 gene therapy for the treatment of osteoarthritis (OA) chronic knee pain; Additional translational data expected before year-end

DELRAY BEACH, FL / ACCESSWIRE / May 16, 2024 / Adolore BioTherapeutics ("Adolore" or the "Company"), a biotechnology company focused on developing breakthrough opioid-free gene therapy treatments for chronic pain, today announced the publication of its manuscript titled, " rdHSV-CA8 non-opioid analgesic gene therapy decreases somatosensory neuronal excitability by activating Kv7 voltage-gated potassium channels,[1]" in the peer-reviewed journal, Frontiers in Molecular Neuroscience.

Roy Clifford Levitt, MD, Clinical Professor at the University of Miami, Principal Investigator and Program Director of the NIH, NINDS, HEAL Award supporting ADLR-1001 development for the treatment of chronic knee pain due to OA, and Founder & Executive Chairman of Adolore BioTherapeutics, highlighted data from preclinical electrophysiological studies of their gene therapy expressing a human carbonic anhydrase-8 variant peptides (CA8*). This manuscript demonstrates that when CA8* expression is increased in nociceptors (specialized pain-sensing neurons), it can attenuate their excitability via a mechanism that involves CA8-induced prolongation of their afterhyperpolarization (AHP) resulting from the activation of cell membrane Kv7 voltage-gated potassium channels. The specificity of the treatment was confirmed using a null-mutant CA8* gene therapy vector and in vitro drug-mediated (XE-991) selective antagonism of the Kv7 channels.

Dr. Levitt, commented, "Kv7 channels remain important analgesic targets. Kv7 voltage-gated potassium channel activators that open these channels and hyperpolarize nociceptors are well-known to produce potent non-opioid-based analgesia in many human chronic pain conditions. Kv7 openers have been successfully translated from animal models to human chronic pain conditions. Based on our findings, we believe that the activation of Kv7 channels by CA8 gene therapy mediated by the reduction of cytoplasmic free calcium explains the analgesia observed through prolonged afterhyperpolarization (AHP) and reduced neuronal excitability. Bolstered by our substantial body of data, we continue to develop our innovative approach to address the significant serious unmet need for safe and effective locally acting pain therapies to replace opioids. Our preclinical data strongly support continued preclinical development toward an IND and clinical studies of ADLR-1001."

Key Highlights

Leveraging its innovative gene therapy vectors expressing CA8* analgesic peptides (ADLR-1001), Adolore is currently advancing two preclinical development programs: ADB-101 for the treatment of patients' chronic pain caused by erythromelalgia, an orphan disease, and ADB-102, their lead program for the treatment of patients with chronic pain caused by knee OA. Based on substantial compelling preclinical data generated to date, the Company is progressing these programs toward IND filings and first-in-human clinical studies.

The Company's lead development program for the treatment of chronic pain in knee osteoarthritis is fully funded by a UG3/UH3 grant awarded to the University of Miami by NIH/NINDS HEAL program to support all formal pre-clinical GLP/GMP/GCP development work through a first-in-human study of ADLR-1l01 in patients expected to commence in 2026.

About Carbonic Anhydrase-8 (CA8*) Gene Therapy

CA8* (variants of naturally occurring human carbonic anhydrase-8 analgesic peptides) gene therapies are a novel class of neuronal calcium channel inhibitors that activate Kv7 voltage-gated potassium channels and are administered locally and long-acting. Oral small molecule pain therapeutics that activate Kv7 voltage-gated potassium channels demonstrated proven analgesic efficacy before they were removed from the market due to severe adverse events related to systemic exposure and their metabolism. CA8* gene therapy provides versatile dosing regimens and routes of administration, including intra-articular, intra-neuronal (nerve block), and intradermal injection. This non-opioid CA8* mechanism-of-action addresses neuropathic, inflammatory, and nociceptive pain, which applies to a broad range of chronic pain indications. These conditions include osteoarthritis, lower back, and cancer pain; diabetes and other forms of peripheral neuropathy, including post-herpetic neuralgia; as well as rare pain conditions such as erythromelalgia, a heritable chronic pain condition, and orphan drug disease.

About Adolore BioTherapeutics, Inc.

Adolore BioTherapeutics, Inc., is a biotechnology company focused on developing novel therapies for treating chronic pain using a revolutionary intra-cellular replication-defective HSV (rdHSV) drug delivery platform that is disease-free, non-toxic, and permits localized peripheral nervous system delivery of proprietary biotherapeutics. This rdHSV gene therapy technology incorporates an established re-dosing strategy and an excellent safety profile. HSV vectors are known for their stability and prolonged gene expression, providing an excellent basis for the long-term treatment of chronic pain conditions. Our best-in-class CA8* programs are long-acting, locally acting gene therapies that are opioid-free Disease-Modifying Anti-Pain therapies (DMAPs) designed to treat many forms of chronic pain.

The Company's current CA8* gene therapy programs are in preclinical development for the treatment of patients suffering from erythromelalgia, a lifelong heritable chronic pain condition representing an orphan drug disease with no approved therapy, and chronic pain due to knee osteoarthritis, affecting a large number of patients that are often treated with opioids due to the lack of good alternatives, thus contributing to the ongoing opioid crisis.

For more information, visit adolore.com.

Forward-Looking Statements

To the extent, this announcement contains information and statements that are not historical, they are considered forward-looking statements within the meaning of the federal securities laws. You can identify forward-looking statements by the use of the words "believe," "expect," "anticipate," "intend," "estimate," "project," "will," "should," "may," "plan," "intend," "assume" and other expressions which predict or indicate future events and trends and which do not relate to historical matters. You should not rely on forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, some of which are beyond the control of the Company. These risks and uncertainties include but are not limited to, those associated with drug development. These risks, uncertainties, and other factors may cause the actual results, performance, or achievements of the Company to be materially different from the anticipated future results, performance, or achievements expressed or implied by the forward-looking statements.

Investor Relations Contact:

JTC Team, LLC Jenene Thomas 833-475-8247 [emailprotected]

SOURCE: Adolore Biotherapeutics, Inc.

Read more:
Adolore BioTherapeutics Announces Publication Demonstrating Carbonic Anhydrase-8 Gene Therapy (rdHSV-CA8 ... - AccessWire

Ocugen, Inc. (NASDAQ: NASDAQ:) discussed significant advancements in its gene therapy programs and provided a financial update during its First Quarter 2024 Financial Results and Business Update call. The company highlighted the clearance of their Investigational New Drug (IND) application by the FDA for the OCU400 Phase 3 limelight clinical trial, with patient screening underway.

The European Medicines Agency (EMA) also deemed the U.S.-based trial acceptable for a future market authorization application. Ocugen revealed progress in their OCU410 and OCU410ST programs, now in Phase 1/2 trials.

Financially, the company reported a net loss of $11.9 million for the quarter, with research and development expenses amounting to $6.8 million. Their cash position stood at $26.4 million as of the end of March 2024.

Ocugen's update during the earnings call reflects a company at a pivotal stage of clinical development, balancing the progress in its innovative gene therapy trials with the financial realities of research-intensive operations. As Ocugen navigates the path towards potential market authorization, its financial health and clinical milestones will be closely watched by investors and stakeholders in the biotechnology sector.

Ocugen, Inc. (NASDAQ: OCGN) has been making strides in its clinical development, as evidenced by the recent FDA clearance for its OCU400 Phase 3 trial. The company's focus on advancing its gene therapy programs is a key factor in its growth strategy. Still, the financial health of Ocugen is a critical aspect that investors are keenly observing. Here are some insights from InvestingPro that shed light on the company's financial status and stock performance:

InvestingPro Data indicates a market capitalization of $437.45 million, reflecting the company's current valuation within the biotech industry. Despite the challenges, the company has shown a significant return over the last week, with a 15.29% increase in its stock price.

This volatility could be of interest to investors looking for short-term opportunities. The revenue growth for the last twelve months as of Q4 2023 stands at an impressive 142.6%, signaling potential optimism for the company's future sales trajectory.

Two InvestingPro Tips that are particularly relevant to Ocugen's current situation include the company's rapid cash burn and weak gross profit margins. With a net loss of $11.9 million reported for Q1 2024 and a decrease in cash and cash equivalents, these concerns are underscored by the fact that analysts do not anticipate the company will be profitable this year.

Moreover, the gross profit margin for the last twelve months as of Q4 2023 was a staggering negative 555.62%, further emphasizing the financial challenges Ocugen faces.

For investors seeking more comprehensive analysis and additional InvestingPro Tips, visiting https://www.investing.com/pro/OCGN can provide a deeper dive into Ocugen's financial metrics and stock performance. Currently, there are over 10 additional tips available on InvestingPro, offering a broader perspective on the company's market position and potential investment risks or opportunities.

To enhance your investment research on Ocugen, consider using the coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription at InvestingPro. This promotion can give you access to valuable insights that may influence your investment decisions regarding Ocugen and other companies in the biotechnology sector.

Operator: Good morning, and welcome to Ocugen's First Quarter 2024 Financial Results and Business Update. Please note that this call is being recorded. At this time, all participants' lines are in listen-only mode. Following the speakers' commentary, there will be a question-and-answer session. I will now turn the call over to Tiffany Hamilton, Ocugen's Head of Corporate Communications. You may begin.

Tiffany Hamilton: Thank you, operator, and good morning, everyone. Joining me on today's call and webcast is Dr. Shankar Musunuri, Ocugen's Chairman, CEO, and Co-Founder, who will provide a business update and an overview of our clinical and operational progress; Michael Breininger, our Corporate Controller, is also on the call to provide a financial update for the quarter ended March 31, 2024; Dr. Arun Upadhyay, Chief Scientific Officer, Head of Research and Development; and Dr. Huma Qamar, Chief Medical Officer will be available to answer questions following the presentation. This morning, we issued a press release detailing associated business and operational highlights for the first quarter of 2024. We encourage listeners to review the press release, which is available on our website at ocugen.com. This call is being recorded and a replay with the accompanying slide presentation will be available on the Investors section of the Ocugen website for approximately 45 days. This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may in some cases use terms such as predicts, believes, potential, proposed, continue, estimate, anticipate, expect, plans, intends, may, could, might, will, should, or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements include, but are not limited to statements regarding our clinical development activities and related anticipated timelines. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission, the SEC, including the risk factors described in the section entitled, "Risk Factors", and the Annual Report we filed with the SEC. Any forward-looking statements that we make in this presentation speak only as of the date of the presentation. Except as required by law, we assume no obligation to update forward-looking statements contained in this presentation whether as a result of new information, future events or otherwise after the date of this presentation. Finally, Ocugen's quarterly report Form 10-Q covering the first quarter of 2024 has been filed. I will now turn the call to Dr. Musunuri.

Shankar Musunuri: Thank you, Tiffany, and thank you all for joining us today. As detailed in our press release, we are excited to discuss the substantial progress of our game changing modifier gene therapy platform by using nuclear hormone receptors to restore homeostasis in the retina, modifier gene therapy has the potential to address multiple inherited retinal diseases as well as diseases larger multi-factorial diseases such as dry age-related macular degeneration through a gene agnostic approach. Just after the close of the first quarter of this year, we announced that the FDA cleared our IND application for the OCU400 Phase 3 limelight clinical trial and the EMA provided acceptability of the U.S. based trial for submission of a market authorization application. I will discuss the significance of these milestones and the potential for OCU400 in greater depth later in the presentation. OCU410 and OCU410ST are now in Phase 1/2 clinical trials targeting geographic atrophy secondary to dry age-related macular degeneration and Stargardt disease, respectively with the clinical updates expected in the third quarter of 2024. Our cell therapy platform is poised to advance with the renovations to our existing facility completed earlier this year. This gives Ocugen the capability to support NeoCart autologous cell therapy manufacturing for personalized Phase 3 clinical material contingent upon adequate funding. Finally, OCU500 will soon advance into a Phase 1 clinical trial via the National Institute of Allergy and Infectious Diseases, NIAID sponsored trial comparing the administration of OCU400 via two different mucosal routes, inhalation into the lungs and as a nasal spray. With three gene therapy candidates to treat blindness diseases in the clinic and the Phase 3 ready cell therapy candidate, we are confident that 2024 will be well weather for Ocugen. We are thrilled to share significant advancements in our leading modified gene therapy candidate OCU400, as it makes remarkable strides in clinical development. The green light from the FDA to begin the Phase 3 clinical trial marks a pivotal milestone as OCU400 becomes the first gene therapy to progress to Phase 3 trials with such a broad retinitis pigmentosa indication. Until now, there has been only one market product to treat one of the 100 gene mutations associated with RP. Now, there is real hope for all RP patients who haven't had a treatment option. Our completion of enrollment and dosing in Phase 1/2 trial demonstrated promising safety and efficacy across various genetic mutations and dosage levels, paving the way for Phase 3 clinical trial. OCU400 has already received key regulatory approvals including orphan drug regenerative medicine, advanced therapy and orphan medicinal product designations from both FDA and the European Commission for treating inherited retinal diseases. These endorsements highlight OCU400's broad therapeutic potential, and we remain on track to meet 2026 BLA and MAA approval targets. We expect to begin dosing patients in the Phase 3 liMeliGhT clinical trial in the second quarter of 2024, which will include 150 subjects across two arms, one with patients affected by raw mutation and one arm that is gene agnostic. Luminance Dependent Navigation Assessment, LDNA, the primary endpoint for the study, focuses on the proportion of responders in treated and untreated control groups, achieving an improvement of at least 2 Lux Levels from baseline in the study acts. The secondary endpoint will be measured by a change in low luminescence visual equity score of 0.3 LogMAR from the baseline. The effectiveness of the treatment will be compared to an untreated control group for the secondary endpoint as well. Additionally, leveraging a dual track strategy, we plan to expand the Phase 3 trial in the latter half of 2024 to include patients with Leber Congenital Amaurosis, LCA contingent on favorable results from Phase 1/2 study. Let me take a moment to discuss the unmet need and underserved market for RP and LCA patients. An estimated 1.6 million people globally are affected by RP and LCA combined in the U.S. and Europe alone, our initial target markets, there are nearly 300,000 total patients. RP and LCA are classified as inherited retinal diseases from a group of heterogeneous disorders that affect the retina. These conditions frequently result in vision impairment and ultimately blindness. By establishing homeostasis in patients affected by these diseases, our aim is to preserve our improved vision by actively sharing our insights at pertinent medical conferences and engaging with advocacy groups, we strive to raise awareness of the desperate need to find a solution for RP and LCA patients, as well as potential of modified gene therapy to revolutionize treatment. To better understand the experience of one of our Phase 1/2 clinical trial patients, I encourage you to watch his video in the patient section of our website. Moving on to our development in the treatment of geographic atrophy, secondary to dry age-related macular degeneration dAMD and Stargardt disease with our OCU410 and OCU410ST programs. These modified gene therapies leverage the nuclear receptor gene RAR Related Orphan Receptor A, RORA aiming to provide a potential one-time therapy for life with a single subretinal injection. OCU410 specifically designed to address multiple pathways implicated in the pathogenesis of dAMD offers a distinct advantage over current treatments that target only one cause of DGA and often require multiple injections per year accompanied by various safety concerns. Our approach with OCU410 is to provide a comprehensive and durable solution, a potential one-time therapy for life. Dry AMD (NASDAQ:) affects about 19 million people in the U.S. and Europe combined, while GA affects about 2 million to 3 million people in the U.S. and Europe a significant market opportunity. In December 2023, we began the Phase 1/2 ArMaDa clinical trial for OCU410. Considerable progress has been made with the completion of dosing in both the first and second cohort, low and medium doses. Following the successful dosing in this cohort, the Data and Safety Monitoring Board, DSMB will convene later this month to evaluate proceeding with the high dose cohort in the ongoing dose escalation phase of the study. After completion of the third cohort, we'll transition into Phase 2 clinical trial, the expansion phase. A key upcoming event for this trial is a clinical update, which is anticipated in the third quarter of 2024. This data will provide initial insights into the safety and efficacy of OCU410. OCU410 Phase 1 study is multi-center and open-label focusing on dose ranging, while Phase 2 is randomized, aiming to expand our understanding of drugs efficacy and safety compared to a controlled untreated arm with patients randomized in a 1:1 ratio across two dosage groups and one control group. Participants must be aged 50 or older, have a best corrected visual equity of approximately 24 letters or more using the ETDRS chart, and have a total geographic atrophy area between 2.5 and 20.5 millimeter square. We now turn to OCU410ST, which received orphan drug designation from FDA for the treatment of ABCA4 associated retinopathies including Stargardt disease. Stargardt affects approximately 100,000 people in the U.S. and EU combined. This modified gene therapy candidate also utilizes the RORA gene in the Phase 1/2 GARDian trial for Stargardt disease is actively enrolling patients. We have completed dosing in the first cohort low dose. In April 2024, the Data Safety and Monitoring Board approved the continuation to the medium dose phase, which we expect will be completed this month. A clinical trial update for OCU410ST is also anticipated in the third quarter of 2024. The Phase 1/2 trial involves up to 42 participants, 30 adults and 12 children, which exhibit mild to moderate disease symptoms, the study uses a 3 + 3 dose escalation design in Phase 1, segmenting subjects into low, medium, and high dose cohorts. Following the dose escalation phase, the trial will transition into Phase 2, the expansion phase. This expansion phase will use a one to one to one design to randomize subjects into two different treatment groups at a varying dose levels or a control untreated group, allowing for a comprehensive assessment of treatments, efficacy across different dosages. Our work across OCU400, OCU410 and OCU410ST affirm our enduring commitment to the success of these modified gene therapies for the benefit of patients faced with the terrible prospect of losing their sight. We are encouraged by the progress in these trials and look forward to sharing further updates as we reach critical milestones and get closer to addressing substantial unmet medical needs. With that, I will now turn the call over to our corporate controller to provide an update on our financial results for the first quarter ended March 31, 2024. Mike?

Michael Breininger: Thank you, Shankar. Our research and development expenses for the quarter ended March 31, 2024, were $6.8 million, compared to $10.2 million for the first quarter of 2023. General and administrative expenses for the quarter ended March 31, 2024, were $6.4 million, compared to $8.3 million during the same period in 2023. Net loss was approximately $11.9 million or $0.05 net loss per share for the quarter ended March 31, 2024, compared to a net loss of approximately $17.3 million, or $0.08 net loss per share for the first quarter of 2023. Our cash and cash equivalents totaled $26.4 million as of March 31, 2024, compared to $39.5 million as of December 31, 2023. As always, we are constantly exploring strategic and shareholder friendly opportunities to increase our working capital. That concludes my update for the quarter. Tiffany, back to you.

Tiffany Hamilton: Thank you, Mike. We will now open the call for questions. Operator?

Operator: Thank you. We will now begin the question-and-answer session. [Operator Instructions]. And your first question comes from the line of Arthur He with H.C. Wainwright. Please go ahead.

Arthur He: Hey, good morning, Shankar and team. Congrats on the progress. So I just had a quick one regarding the 400 history study. For the gene agnostic arm, is there a minimum number of patients for each specific gene mutation to meet the requirement for the BLA application?

Shankar Musunuri: Hey Arthur, good morning. I'll let Arun take the response.

Arun Upadhyay: No. We have not pre-specified any specific number of patients for each mutation, but our approach is going to be include as many mutations as possible in the gene agnostic count.

Arthur He: Okay. Thanks, thanks. Thanks for the color. And also my second question is regarding the 200. Could you give us more color on the status and what's your effort to get the clinical holder lifted as soon as possible?

Shankar Musunuri: Arun?

Arun Upadhyay: Yes. So we are working on addressing the CMC question we received from FDA and we are planning to address that ASAP and then continue the development of OCU200 further.

Operator: Your next question comes from Robert LeBoyer with Noble Capital Markets. Please go ahead.

Robert LeBoyer: Good morning and congratulations on all the progress during the quarter. My question has to do with the Phase 3 trial coming up and you had mentioned some milestones to start. Are there any timeframes or any particular milestones that we can look forward to in terms of accrual or projected time for data release?

Shankar Musunuri: Yes, Robert, I let Dr. Qamar answer the question.

Huma Qamar: Hi, thank you for your question. In terms of the milestones, as you're aware that OCU400 has recently got approval. We're currently screening patients and we will be continuously updating the market with dosing updates very soon.

Robert LeBoyer: Okay. Is there any projected time for the first data release?

Huma Qamar: For OCU400, Phase 3 or Phase 1/2.

Robert LeBoyer: Either actually?

Huma Qamar: Okay. So Phase 1/2, we have already updated that, continuous updates will be coming in the next quarter as well. And as soon as we receive more data on the LCA patients. For OCU400 Phase 3, it will be, there is no time frame as it will be periodic. And we will be giving periodic updates to the market as soon as it's available.

Shankar Musunuri: And it's a -- Robert, I think this is a controlled clinical trial for Phase 3. So until the study is done, we cannot reveal any information to the market on the efficacy portion of it.

Robert LeBoyer: Sure. That's --

Shankar Musunuri: We'll provide periodic updates, as Dr. Qamar is saying, on recruitment and where the trial is going. And also give markets an update about on track for our target approval for BLA and MAA in 2026.

Robert LeBoyer: Okay. Yes, that's probably as much as anyone can say for a rare disease like this. So thank you.

Operator: Your next question comes from the line of Daniil Gataulin with Chardan. Please go ahead.

Daniil Gataulin: Yes. Hey, good morning, guys. Thank you for taking the question. Also a question on OCU400 and Phase 3, so you mentioned that as part of deal track strategy, you will be expanding Phase 3 to include patients with LCA pending the Phase 1 LCA data. I just wanted to clarify, this will be added as a new arm in the trial or if it will be a separate standalone trial. And I guess the second part of the question is, what data from LCA Phase 1/2 would you consider favorable that would give you a go ahead decision for these patients. Thank you.

Shankar Musunuri: Arun will take the call.

Arun Upadhyay: Thanks, Shankar. Yes. So based on the Phase 1/2 study outcome, our plan is to have the separate trial for the LCA because it's different disease. So once we have the data available, we'll be engaging with agency. And based on the agency recommendation, we will initiate the trial accordingly.

Daniil Gataulin: Okay. Got it. Thank you. And in terms of the Phase 1/2 data threshold, what would you consider favorable to advances into Phase 3?

Arun Upadhyay: Primarily the safety and efficacy.

Operator: This concludes the Q&A portion. I will now turn the call back over to Chairman and CEO, Dr. Shankar Musunuri.

Shankar Musunuri: Thank you, Operator. Our efforts in the first quarter of the year evidence the importance of our gene therapy programs and the need to operate the business to ensure their success. We are opportunistic about Ocugen cell therapy and vaccine platforms, knowing that these technologies have great therapeutic and financial potential, and are pursuing partnerships to support our entire pipeline. We look forward to what this quarter and the rest of the year holds for the company, our people, and the patients we serve. Tiffany?

Tiffany Hamilton: Thanks, everyone. Bye.

Operator: Ladies and gentlemen, that concludes today's call. Thank you all for joining. You may now disconnect.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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Earnings call: Ocugen reports progress in gene therapy trials and Q1 financials - Investing.com

Voyager Therapeutics, Inc. (NASDAQ:) has announced significant progress in its gene therapy programs and a new strategic collaboration during its latest earnings call.

The company has received Investigational New Drug (IND) clearance for its anti-tau antibody VY-TAU01 for Alzheimer's disease and is poised to begin dosing in a human trial soon.

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Operator: Good afternoon, and welcome to Voyager Therapeutics First Quarter 2024 Financial Results Conference Call. [Operator Instructions] Please note that today's conference is being recorded. A replay of today's call will be available on the Investors section of the company's website approximately two hours after completion of this call. I would now like to turn the call over to Trista Morrison, Chief Corporate Affairs Officer.

Trista Morrison: Thank you, and good afternoon. We issued our first quarter 2024 financial results press release this afternoon. The press release and 10-Q are available on our website. Joining me on today's call are Dr. Al Sandrock, our Chief Executive Officer; Dr. Toby Ferguson, our Chief Medical Officer; and Dr. Todd Carter, our Chief Scientific Officer. We will also be joined for the Q&A portion of the call by our Chief Operating Officer and Principal Financial (NASDAQ:) Officer, Robin Swartz. Before we get started, I would like to remind everyone that during this call, Voyager representatives may make forward-looking statements, as noted in Slide 2 of today's deck. These statements are based on our current expectations and beliefs. They are subject to risks and uncertainties, and our actual results may differ materially. I encourage you to consult the risk factors discussed in our SEC filings, which are available on our website. And now I will turn the call over to Al.

Alfred Sandrock: Thank you, Trista, and good afternoon, everyone. Please turn to Slide 3. I'd like to start by thanking the Voyager team for their dedication to creating transformative genetic medicines. We made tremendous progress advancing these medicines in the first quarter. We just announced that we have obtained IND clearance for our anti-tau antibody, VY-TAU01, for Alzheimer's disease, and we expect to announce the dosing of the first subject in a single ascending dose trial in healthy volunteers in the coming week or so. Our gene therapy pipeline also advanced during the quarter with development candidates selected in the GBA1 and Friedreich's Ataxia programs partnered with Neurocrine. These programs, along with our wholly owned SOD1-ALS program, are advancing towards IND filings in 2025. In March, we appointed Dr. Toby Ferguson as our Chief Medical Officer. Toby is an exceptional biotech executive with deep experience advancing novel therapies for CNS diseases. This includes tofersen, the first genetically targeted therapy to be FDA-approved for SOD1-ALS and the first treatment to receive accelerated approval based on plasma neurofilament light chain response. Toby has hit the ground running, and we look forward to his leadership of our emerging clinical portfolio. In January, we announced an expansion of our relationship with Novartis through a new strategic collaboration and capsid license agreement to advance potential gene therapies for Huntington's disease and spinal muscular atrophy. This agreement, together with the public offering we completed in January, brought $200 million of total consideration to Voyager in the first quarter. This bolstered our balance sheet and extended our runway into 2027, and we expect that it will enable us to achieve multiple clinical data readouts. Finally, we presented a robust set of data at the recent AD/PD and ASGCT meetings, including data on our multiple tau-targeting programs in our second-generation capsids. Given this significant progress, we believe Voyager is emerging as a leader in neurogenetic medicine. Our pillars of value are summarized on Slide 4. First, we have a strong pipeline of four wholly owned and 13 partnered programs with the first expected to enter clinical trials in the coming weeks and the potential for three more to follow next year. Second, we have an industry-leading platform designed to overcome the delivery challenges inherent to CNS gene therapies. Our TRACER platform enables us to create novel capsids that, following IV delivery, harness the extensive cerebrovasculature to enable widespread payload distribution across multiple brain regions and cell types. These capsids have demonstrated translatability in multiple species. And have enabled the selection of multiple development candidates in our wholly owned and partnered gene therapy programs. Third, we have blue-chip partnerships anchored by TRACER's potential to transform the treatment of CNS diseases. In addition to Neurocrine, our partners include Novartis and Alexion (NASDAQ:). In total, our partnered programs could generate up to $8.2 billion in longer-term milestone payments. Finally, we continue to explore the potential to leverage receptors we have identified to shuttle nonviral genetic medicines into the brain. Ultimately, we aim to expand from gene therapy and antibodies into other modalities of neurogenetic medicine, broadening our impact. With that, I'll turn the call over to Toby.

Toby Ferguson: Thank you, Al, and good afternoon. Please turn to Slide 5. This slide summarizes the four wholly owned programs and our 13 partnered programs that Al mentioned earlier. Part of what attracted me to Voyager is that these programs are focused on targets validated by human biology and human genetics. As a drug developer and former practicing neurologist, I've dedicated my career to improving the lives of people living with neurologic diseases. And while our understanding of the genetic and biological basis of CNS diseases has advanced considerably, translating that understanding to new therapies has been hindered by challenges with crossing the blood-brain barrier. I believe that the progress that Voyager has made in this regard is transformative and provides an unprecedented opportunity to redefine the treatment of CNS diseases. I'm delighted to join the company as we prepare to enter the clinic and realize the full potential of our pipeline of neurogenetic medicines. Turning to Slide 6. I want to focus for a moment on two of our wholly owned programs that target tau. We believe tau is a critically important target for the treatment of Alzheimer's disease. The presence of tau pathology in the brain is a cardinal feature of AD. Further, the spread of tau pathology through the brain closely correlates with clinical decline and can be visualized with tau PET imaging. Importantly, recent third-party clinical data generated using an intrathecally administered tau ASO have shown that reducing tau was associated with favorable trends on clinical outcomes. As Al mentioned, we're excited to announce that VY-TAU01, our anti-tau monoclonal antibody, obtained IND clearance. And we look forward to announcing, in the coming weeks, the initiation of a single ascending dose trial in healthy volunteers. I'll talk more about that trial in a moment. First, a bit of background on this program. VY-TAU01 aims to inhibit the cell-to-cell spread of the extracellular forms of pathological tau in the brain. In contrast to third-party anti-tau antibody approaches that have targeted the intrinsical and have been unsuccessful in the clinic. VY-TAU01 targets the C-terminal epitope of pathological tau. We believe the epitope matters. In a preclinical in-vivo model of tau spread, the myriad surrogate of VY-TAU01 inhibited tau spread by approximately 70%, with internally directed antibodies have no significant effect. This indicates the negative predictive value of this model. We look forward to establishing whether or not it has positive predictive value as we advance into clinical trials. Importantly, and in parallel, we are progressing a tau silencing gene therapy approach intended to inhibit the production of tau protein. This program deploys a tau-targeted SRNA packaged into an IV administered TRACER capsid. Using this approach, we've demonstrated robust reductions and tau mRNA and protein across the brain by a single IV administration and mice expressing human tau. We believe this program has the potential to provide a transformative single-dose treatment for Alzheimer's disease. We anticipate filing an IND in 2026. Turning to Slide 7. As I mentioned, we anticipate the announcement dosing of the first subject in our single-ascending dose trial, VY-TAU01 in the coming weeks. This will be a single-site study in which we expect to enroll approximately 48 participants. The primary aim of the trial is to generate initial safety and PK data that will form a subsequent multiple ascending dose trial. We expect to conduct the multiple ascending dose trial participants with early Alzheimer's disease. We expect to initiate this trial next year and generate initial tau PET imaging data in 2026. That has the potential to show slowing of tau spread. Turning to Slide 8. In addition to our programs targeting tau, we are also advancing three gene therapy programs for which we expect to file INDs next year. They include our wholly owned SOD1-silencing program targeting the genetic cause of SOD1-ALS, the Neurocrine partnered GBA1 gene replacement program targeting the genetic cause of Friedreich's Ataxia. And the Neurocrine partnered GBA1 gene replacement program. Mutations in GBA1 represent both one of the most common genetic cause of Parkinson's disease as well as the cause of other GBA1-related diseases. Each of these programs leverages an IV-administered, blood-brain barrier, TRACER capsid and has the potential to provide a single dose disease-modifying treatment. We look forward to advancing these promising suite of programs in the clinic. With that, I'll turn the call over to Todd.

Todd Carter: Thanks, Toby. Please turn to Slide 9. In support of our advancing gene therapy pipeline, we were pleased to present a robust set of data on the potential clinical translatability manufacturing and overall performance of our TRACER capsids at the American Society of Gene and Cell Therapy Annual Meeting last week. Voyager scientists presented a total of 12 abstracts at the meeting, and I'd like to review a few highlights. First, we presented new data on our second-generation IV-delivered TRACER capsids. This session was standing room only with a line out of the door and down the hall, which I think speaks to the high level of interest in novel capsids engineered to cross the blood-brain barrier. Our second-generation capsids should further enhance blood-brain barrier penetrants and reduced liver expression compared to our own first-generation TRACE-derived capsids. These second-gen capsids demonstrated a robust transduction of 50% to 75% of cells across diverse brain regions, with upwards of 95% transduction in certain key cell types, such as Purkinje neurons, at a clinically relevant dose of 3x10 13 vector genomes per kilogram. This also included transduction of 98% of dopaminergic neurons at the substantia nigra and over 80% of spinal motor neurons. Importantly, our TRACER capsids have enabled selection of development candidates for the three lead gene therapy programs that Toby distribute. We presented data at ASGCT on VY-9323, our wholly owned SOD1-silencing gene therapy, which uses a second-generation TRACER capsid. The data demonstrated that a single IV dose of VY-9323 at the 3E13 vector genomes per kilogram dose reduced SOD1 mRNA by up to 80% in spinal cord motor neurons in nonhuman primates. Finally, as part of our strategy to mitigate the risks of developing TRACER capsid-derived product candidates and maximize their probability of success in the clinic, Voyager has been working to identify the receptors that mediate the delivery of these capsids into the CNS and confirm their expression in humans. At ASGCT, we identified tissue nonspecific alkaline phosphatase, or ALPL, formerly known as receptor X, as the highly conserved receptor expressed on brain vasculature that mediates the delivery of TRACER capsids VCAP-101 and 102 across the blood-brain barrier. These capsids bind human, primate and neurine ALPL isoforms, further strengthening our confidence in the clinical potential of TRACER capsids. More broadly, these data establish ALPL as a novel brain delivery shuttle. And we are exploring the opportunity to leverage this receptor to deliver multiple therapeutic modalities across the blood-brain barrier. We look forward to sharing these data in the future. Collectively, the data package we presented at ASGCT represents the most extensive validation to date of the potential clinical translatability of our TRACER capsids. And we look forward to evaluating their ability to transform the course of a broad range of neurological diseases. I will now turn the call back over to Al.

Alfred Sandrock: Thanks, Todd. Turning to Slide 10. You can see Voyager has had an incredibly strong start to the year. As I mentioned before, this would not be possible without the hard work and dedication of our employees. I know many of them are listening, so I want to say, again, thank you. With a robust slate of upcoming clinical milestones, a maturing partnership portfolio and cash runway into 2027, we believe Voyager is poised to drive significant value over both the near and long term. With that, we will open the call for questions. Operator?

Operator: [Operator Instructions] Our first question comes from the line of Joon Lee of Truist Securities. Your line is now open.

Mehdi Goudarzi: Hi. Good afternoon and congrats on the great progress. This is Mahdi on for Joon. Last week at ASGCT, you showed great data related to a Receptor X, ALPL. So could you please elaborate on the ways that you are planning to use this knowledge for delivery of the other modalities to CNS? Specifically, do you think antisense oligos and LMPs could see similar levels of transcytosis seen with AAVs? Thank you.

Alfred Sandrock: Yes. Thanks for the question. This is Al Sandrock. So briefly, we know that these receptors mediate the transport of these very large AAV capsids across the BBB. And what we're going to do now is to make ligands against the receptor, conjugate them to various macro molecules to see if we can get them to cross the BBB. We believe by transcytosis. And the range of molecules we could look at include protein therapeutics as well as oligonucleotides and we're progressing those experiments as we speak. Todd?

Todd Carter: Thanks, Al. So we are looking at a variety of different modalities. As Al indicated, we're looking at antibodies, oligos, other sorts of proteins. Al mentioned that we're in the process of identifying ligands, and we have identified some ligands. And we're looking forward in the future to hopefully sharing some of those data when we're ready to.

Mehdi Goudarzi: Appreciate it. Thank you.

Operator: Thank you, one moment for our next question. Our next question comes from the line of Jack Allen of Baird. Your line is now open.

Jack Allen: All right, thanks so much for taking the questions and congratulations on the progress. I wanted to ask about some of the work you presented at ASGCT around the optimization of manufacturing. How are you thinking about optimizing manufacturing before you bring candidates into the clinic with your gene therapies? And what do you expect the benefits will be as it relates to COGS of your more potent gene therapies as well?

Alfred Sandrock: Well, I'll start and I'll ask Todd to help here. So we're going to be planning to use HEK293 cells to manufacture these products. We have a very robust internal technical operations team and part of the development candidate selection, we assess manufacturability, both as well as the downstream as well as upstream processes. And of course, we're going to be verifying percentage of full capsids, partially full capsids as well as anti-capsids. And so we assess manufacturability as part of the development candidate selection. Todd?

Todd Carter: So as Al mentioned, we do have that manufacturability is a key component. It's actually part of our whole assessment of our novel capsids as we identify them. In each case, the payload can make a difference so that for any given program, we have to establish the manufacturability with the capsid in question and the particular payload for the disease indication as well. All that goes into our assessment, and we begin with research-grade material. And then once we develop the settlement candidate, then we proceed to the process development to move that into manufacturability. You also asked a question about COGS. And of course, it's not exactly necessarily a 1:1 trade-off. But you can imagine, as we're looking with our novel capsids that have a potency approximately about an order of magnitude greater than what the dose is currently used in the clinic, we expect to see substantial savings because we need much less material to deliver the same level or actually greater levels of delivery to the CNS than the conventional capsids.

Alfred Sandrock: Yes. And I would just add, Jack, that in addition to the lower doses, what we note is that we're going to transfer the process to a CDMO for manufacturing. And the CDMO world has really come up to speed in terms of scalability. We're now seeing scale. And every time you increase the scale, we actually reduce the cost. So -- and at the pace that it's growing, we expect to see continued advancement in that area as well.

Jack Allen: That's great. Can I just ask one final question on that? I know we haven't really gotten this far in the gene therapy space, but how does the shelf life of these products play a role when you think about the commercial applicability of this manufacturing scale here?

Alfred Sandrock: Shelf life. Wow, that's a question I hadn't thought about yet. But anybody on here has an answer to that?

Todd Carter: So we do assess the shelf life. And of course, for a gene therapy, we typically would need to require to be held at particular cold temperatures. All that is part of the evaluation for stability, both short term and long term. So that's an important part of our manufacturability and our process development.

Jack Allen: Thanks, Todd. Thanks so much. Congrats on the progress.

Todd Carter: Thank you.

Operator: Thank you. One moment for our next question. Our next question comes from the line of Patrick Trucchio of H.C. Wainwright & Co. Your line is now open.

Patrick Trucchio: Thanks. Good afternoon and congrats on all the progress. Just a couple of follow-up questions for me. The first is, I'm wondering if you can discuss any potential read-through that you'll be looking for from the advisory committee meeting on June 10 regarding donanemab in Alzheimer's disease to your Alzheimer's programs, and then separately, just a follow-up on the ASGCT data, specifically the data in human tau mouse model that showed reduction in tau mRNA levels up to 90% and 50% to 70% reduction in tau protein. Can you tell us how this data compare with prior-generation capsids and how the data may support advancement of tau silencing gene therapy IND in 2026?

Alfred Sandrock: Thanks, Patrick. I'll answer the first question, and maybe I'll ask Todd to answer the second one. On the first question, well, it will be a very interesting advisory committee. We do have a vectorized anti-amyloid program in our pipeline as well. So we're watching that advisory committee with interest. I think a lot of the questions might be unique to donanemab and may not pertain very much to our program. Nevertheless, there might be some important features. For example, what are the outcome measures that are going to be important for approval. And of course, that's always something that we need to keep an eye on. But that's down the line for us. We have to get into the clinic first. But -- so that's the kind of thing we may be paying attention to, but it will be an interesting meeting. Todd?

Todd Carter: So on our tau knockdown program, the data that you're referring to, we showed some at AD/PD and also at ASGCT. The knockdown in the mouse was with a mouse-capable capsid. So we used that really to assess the payload and also to hopefully demonstrate, and we feel that we did, that it's -- the payload has the ability to knock down tau, both mRNA and protein at a clinically relevant dose. So we think that, that's what we showed in those mouse studies. With regard to the payload, we were able to see a well-tolerated and safe dose in those animals, delivering sufficient vector to the brain. And that we saw a quite remarkable knockdown of the mRNA in those animals. So it's building that proof of concept for the knockdown, and we're moving forward now and evaluating the novel capsids for nonhuman primates and humans with the payloads in question. So we're looking forward to sharing more of that in the future.

Patrick Trucchio: Great, thanks so much.

Operator: Thank you. One moment for our next question. Our next question comes from the line of Philip Nadeau of TD Cowen. Your line is now open.

Philip Nadeau: Good afternoon. Congrats on the progress, and thanks for taking our questions. First, a couple on VY-TAU01. Have you disclosed what doses you are going to be exploring in the single ascending dose trial? And can you talk a little bit how you're going to use the pharmacokinetic data that you gathered to determine what doses should be explored in the MAD trial. How will you guess -- extrapolate from the single dose to the multiple doses and particularly for brain peripheral versus maybe crossing the blood-brain barrier? Thanks.

Toby Ferguson: Thanks for the call. This is Toby. We haven't disclosed our doses, but broadly speaking, we've examined our candidates in our preclinical model of tau spread, where we've shown that we see reduction by about 70% of the spread of pathologic tau in our mouse models. In addition, we've done preclinical work in primates, I think -- and fundamentally, this is a single ascending dose study in about 48 patients over multiple cohorts. We expect, based on that data, to get both, of course, safety information as well as PK information on BBB that we think we can appropriately translate into doses for the MAD in mid-2025, whether we want to aim to estimate sort of the underlying exposures needed to get appropriate exposure in the brain to treat this.

Alfred Sandrock: Yes. And Phil, I may want to add that I mean, based on our preclinical studies, including an NHP, we don't want to anticipate any major surprises. This is likely to be very similar to other monoclonal antibodies and, therefore, the brain-to-plasma ratio will be in the 0.1% to 0.5% range. And as Toby said, we know the exposures that we need to get into the brain to inhibit tau spreading in the model that we used to choose the antibody. So that will give you some idea, and of course, we do expect that trial to inform the dose range in the upcoming multiple ascending dose trial.

Philip Nadeau: That's really helpful. And then second, on the frataxin candidate, would you be able to disclose anything new about the candidate that was chosen? And anything notable that you'd be willing to tell us about what differentiated that candidate from the others?

Alfred Sandrock: Well, so that's a Neurocrine program. And so we're -- we don't want to disclose these things. Suffice it to say that the development candidate we had a set of criteria for the capsid as well as the capsid-plus-payload combination, both in terms of its mechanistic effects in animals as well as manufacturability. And we'll ask Neurocrine to answer your question.

Philip Nadeau: Fair enough. Thanks for taking our questions. Congrats, again, on the progress.

Alfred Sandrock: Thank you.

Operator: Thank you. One moment for our next question. Our next question comes from the line of Ry Forseth of Guggenheim Securities. Your line is now open.

Ry Forseth: Hi. This is Ry from Debjit's team at Guggenheim. From the ASGCT data, now with gen-3 technology maturing, where you're able to navigate preexisting neutralizing antibodies, how are you framing the market opportunity expansion given the preclinical profile you're seeing to date?

Alfred Sandrock: I'll start, and maybe Toby or Todd can add. But yes, so you noticed that our -- one of our posters that we are looking -- we're leveraging TRACER to see whether we can make modifications to capsids that affect immunogenicity. And by that, what we're doing is we're looking to see whether preexisting antibodies in humans can affect combined to the capsids, actually, and therefore, affect how they perform in the clinic. And so obviously, if we can find novel capsids that can evade, if you will, the preexisting antibodies, more patients would be available for treatment. In addition, I would note that we and others are going to be evaluating ways in which we can lower preexisting antibody levels. And as you know, several other companies are investigating the use of various enzymes that can degrade preexisting antibodies. So that will be another way to achieve essentially the same thing, which is to try to get more patients to be eligible for our treatments. Toby?

Toby Ferguson: I would agree with Al. I think the only point I would make in addition is that this may be particularly important in adult populations that may be select pediatric populations with there's not as much of a concern. That may represent some opportunities.

Ry Forseth: Fascinating work.

Operator: Thank you. One moment for our next question. Our next question comes from the line of David Hoang of Citigroup. Your line is now open.

David Hoang: Hi there, congrats on the progress and thanks for taking my question. I guess first, I just wanted to ask about how you think about the anti-tau antibody fitting into the treatment landscape of Alzheimer's as compared to the tau silencing gene therapy that you also have? What could be the advantages versus maybe disadvantages of each of those modalities? And then maybe just a second question. Have you interrogated -- or to what extent have you interrogated other transporters besides ALPL for crossing the blood-brain barrier? And if you've done that work, how did ALPL compare to other potential transporters? Thanks.

Alfred Sandrock: So I'll start on the first question. And then Toby, I'll ask Toby to help with that one. And then, Todd, maybe you can answer the second question. So anti-tau versus tau silencing. So very different approaches. Anti-tau, we expect to bind the extracellular forms of tau. And as Todd said earlier, what we're trying to do is to block the spread of pathological tau, which we demonstrated quite nicely in an animal model where we inject human pathological tau into the animals and look at spread. The tau knockdown is a very different -- so that's an antibody that will need to be given on a regular basis. probably on the order of every month or so, every four weeks, we anticipate. The tau knockdown is a gene silencing that will be done by gene therapy. So that would be a once and done, essentially. And it decreases the expression of all forms of tau. It's akin to others have used an antisense approach to decrease the expression of tau, so it's a different mechanism of action. And we'll have to see -- the first thing is which one works the best. And then we'll see whether or not it can be done once and done or whether it will need to be regular infusions. Toby?

Toby Ferguson: Thank you, Al. I think what I would add is -- in concert, we fundamentally, at this point, don't have enough clinical data to understand the potential completeness of treatments with either with any of these modalities. I think first and foremost, the most important point is determining which ones work. and we're excited to test have two opportunities to test taus in our programs, both the antibody program and the knockdown program. I do think moving forward, we'll need to look and try to understand the combination as well, and that is the potential to be investigated in the future.

Alfred Sandrock: And Todd, do you want to take the second question?

Todd Carter: Sure. The second question, just to recap, would be the evaluation of ALPL and other in the context of other potential shovels or shovel targets for BBB delivery. So we absolutely would need to evaluate ALPL in the context of other receptors, such as transferrin and there are a few others. While we're not in a position to share any data today, you can imagine that anything we would choose to move forward would need to perform at least as well as the existing BBB transporters and so that would be part of our assessment and our evaluation, and looking forward to, hopefully, in the future, being able to share more about our work in that area.

Alfred Sandrock: And then, Todd, I thought -- I think maybe David was also asking whether we have other receptors as well and how we're looking at those perhaps and comparing them to ALPL.

Todd Carter: So we have identified some other receptors. We have multiple capsid families that target different receptors. And so in terms of our novel receptor discovery, we have identified a few, and we are in the process of evaluating all of them for nonviral delivery.

Operator: Thank you. One moment for our next question. Our next question comes from the line of Jay Olson of [Oppenheimer]. Your line is now open.

Unidentified Analyst: Hi. This is Chung on the line for Jay. Congrats on the progress. Maybe a 2-part question on the ALPL program you've disclosed. Just first, I'm just wondering if you can talk about the expression variability of ALPL in humans and maybe whether the expression may change over time with aging or with some underlying diseases. And secondly, for the development of other therapeutic modality or delivery modality using ALPL, are you planning to do that internally or through collaborations?

Alfred Sandrock: So Todd will answer the first question. I'll answer the second one.

Todd Carter: So on the first question, differences are changes of the ALPL expression. So we can look at different genetic variation of ALPL and we're in the process of doing that. In terms of the expression level, we know that ALPL is expressed at a quite robust level starting at birth from the databases and also from internal work on in preclinical models. That expression goes up a bit with age. So if anything, we might expect even better delivery in older populations, but all the evidence to date suggests that the level of expression in the vasculature is quite robust, even starting at a very early age.

Alfred Sandrock: Yes. And in terms of the second question, we do expect to be doing a lot of the work internally, and we have already started to do that. But we're always looking for collaborations that can enhance that discovery and development efforts. So stay tuned. SP1. Yes, thank you so much.

Operator: Good afternoon. Thank you. One moment for our next question. Our next question comes from the line of Sumant Kulkarni of Canaccord Genuity. Your line is now open.

Sumant Kulkarni: Nice to see all the progress and thanks for taking our questions. I guess these are welcome questions for Dr. Toby Ferguson. So on your SOD1-ALS program, given the limited patient population and because tofersen is on the market already, do you expect to involve tofersen in preclinical work? And how do you expect eventual trial recruitment to play out for why SOD1-ALS program?

Toby Ferguson: Thanks for the question. So I think. Fundamentally, I think, so first one obviously is approved, and I think it's approved on the basis of a biomarker, particularly neurofilament, and it is indeed a disease-modifying therapy for ALS administered once interest. I think what I'd point to you on our programs...

Alfred Sandrock: Once a month.

Toby Ferguson: Excuse me, Once a month, excuse me. What I'd point you in our programs is that we did discuss recently at ASGCT really the transduction data. I think I'd highlight that at the start. In motor neurons, we saw 80%, 93% transduction and up to 6% to 8% in cortex when we looked at second-gen VCAP capsids. So that's really quite important. And that was with an intravenous administered dose. In the context of our VY-9323 program, we saw a 72% reduction of mRNA and SOD1 mRNA and 80% present in the core. So I think, fundamentally, we'll take some lessons learned from tofersen, particularly around the biomarkers. We'll apply these to our clinical development program. We think this provides us an opportunity to understand not only the potential proof of concept in people with ALS, but also the potential importance for our TRACER platform as a whole.

Alfred Sandrock: And Sumant, maybe I'll add that if we look forward to the potential approval of our SOD1 gene therapy, I'll note that in the case of SMA, SPINRAZA was approved a couple of years prior to Zolgensma. And the two are used sort of in some ways concurrently in the real world. So for example, we're aware that many patients can get treated with Zolgensma right after birth. And then if necessary, they add SPINRAZA. And so taking a gene therapy doesn't preclude continued treatment with the person when necessary. And so I think if we take a page from the SMA story, we could see both products to person as well as, hopefully, our SOD1 gene therapy being used together essentially by physicians to treat their patients optimally.

Sumant Kulkarni: Got it. And as a quick follow-up, what percentage reduction on NFL is considered clinically relevant in the SOD1-ALS context? We've seen the data that Biogen (NASDAQ:) had. But what would be considered clinically relevant, I guess, for what's out there already and for a gene therapy?

Toby Ferguson: So I think we've all seen the reductions that Biogen has highlighted with neurofilament reductions. I think, fundamentally, what a clinically relevant reduction is not clear. But I guess the points I would highlight is it has to be substantially greater than the variability of your assay and biologic variability and that the tofersen data does inform the magnitude of needs of reduction.

Operator: Thank you. One moment for our next question. Our next question comes from the line of Yanan Zhu of Wells Fargo Securities. Your line is now open.

Yanan Zhu: Great, thanks for taking our questions and congrats on the progress. So first, I was wondering about the ALPL receptor, is there expression of this receptor on any other tissue? And how does that potentially impact or not impact a brain delivery drug in terms of delivery into other tissues?

Todd Carter: Sure. This is Todd. I can take that question. So thank you for it. So the ALPL is expressed across the vasculature and the entire body and in some other cell types. However, what we think is going on is that AAV capsids, AAV gene therapies do not need to harness these other mechanisms to get into other tissues. But the blood-brain barrier is blocking for most conventional capsids or all conventional capsids to get into the brain. So the harnessing of ALPL to cross the blood brain barrier is really only important to get into the brain. What we also see is that the use of ALPL is giving us some significant cross-species activity. So it provides us not only with in-vitro evidence of cross-species activity in transcytosis assays, but also in vivo cross-species activity, which we see in multiple species. So we have examples of four species: African green monkeys, cynomolgus macaques, marmosets and mice. And ultimately, what we're able to see is that at relatively low doses, we're able to achieve these kinds of high delivery in transduction that Toby mentioned. 80% of motor neurons in the spinal cord, 95% in Purkinje neurons and 98% of dopaminergic neurons in the substantia nigra. And then finally, I'll just comment that with regard to the tissue and cell delivery, what we're seeing is a significant de-targeting from the liver. That's probably not specifically driven by ALPL or hypothesis is that's based on other characteristics of the capsid. But we see it quite substantially targeting human liver with the simultaneous increase in delivery into the CNS.

Yanan Zhu: Great. That's very nice to hear. And also at ASGCT, there are quite a few presentations on BBB penetrating capsid work from different industry players. And some of them also begin to touch upon receptors. I think you might have started a trend. Just wondering, after taking a survey of the landscape, how do you feel of your BBB capsid and where it stands in the landscape?

Alfred Sandrock: Maybe I'll start and Todd will complete the answer. But -- so listen, it's great to see that a lot of people are finding what we found years ago. And that -- yes, I mean, I think -- I think what we're seeing is that I think the world appreciates the need for new capsids that cross the blood-brain barrier, so as to improve delivery. And look, the competition is heating up. I would say that we're very proud of our capsids. As Todd said, we get the key cells that are relevant for the diseases of interest. We get 80%, 90%, as Todd just mentioned, of cells transduced at relatively low doses of 3E13 VGs per kg, and we have demonstrated in vivo multiple cross-species experiments have been done with three different nonhuman primate species as well as mice, knowing the receptor is also very helpful. So -- and then look, we have already selected three development candidates, two with our partners at Neurocrine and one are wholly owned, and we expect to be in the clinic soon. So Todd?

Todd Carter: I think you captured everything that I was going to say, Al. And I do think the important point in addition to the cross-species activity is, over the past quarter or so, those three candidates that we're moving into the clinic. We think the next step is really the clinic.

Yanan Zhu: Got it. Very helpful. Thank you.

Operator: Thank you. One moment for our next question. Our next question comes from the line of Laura Chico of Wedbush. Your line is now open.

Laura Chico: Good afternoon and thanks very much for taking the questions. Just one housekeeping question. I believe there was one milestone payment that was triggered in the second quarter. But curious if you could just kind of elaborate or if you've disclosed any additional milestones that we should be watching out for over the remainder of 2024? And then I have a quick follow-up for you.

Robin Swartz: This is Robin. Thank you for the question. So we were very pleased with the advancement of our Neurocrine programs. and the achievement of the DC milestone. However, we don't provide guidance on potential future milestone payments across the 13 partner programs. It is also important to note that further milestone payments are not included in our cash guidance, which is into 2027.

Laura Chico: Okay. Thank you very much. And then in terms of the follow-up, obviously, there's a lot of discussion on the ALPL receptor data from ASGCT but I'm wondering if you can just talk, perhaps, Al, strategically, what would happen in terms of the direction of focus for Voyager with the VY-TAU01 data. If that reads out positively in the patients, what does that mean strategically in terms of the focus? Obviously, that's -- as an antibody right now, but how does that shift or change the focus on other TRACER programs, assuming success there?

Alfred Sandrock: Well, Laura, thanks for the question. I think what you're implying is that we would have a choice of whether we proceed with the VY-TAU01 program as an intravenous antibody or whether we could vectorize the antibody. We actually do have that option. We may actually do both, in fact. I've always been thinking, though, that strategically wouldn't make sense for a Voyager, a small company like us to try to go into Phase 3 or commercialize in Alzheimer's disease. It's too large, too expensive. So we've always thought we will likely get a partner if the VY-TAU01 program is positive. But we -- but I do think that the option to potentially vectorize a once-and-done antibody could be of high interest to us, and we'll make that decision when the time comes.

Laura Chico: Thank you very much.

Operator: Thank you. One moment for our next question. Our next question comes from the line of Joon Lee of Truist Securities. Your line is now open.

Joon Lee: Following Laura's question, last week, in ASGCT, [indiscernible] suggested in some of the presentations that compared to capsid and inserted vectorized antibodies, the expression of -- sorry, capsid and inserted vectorized antibodies had a lower expression. Do you expect the same for your vectorized antibody platform in general and specifically for the Alzheimer's antibody. And if could you share any plans that you have for a preclinical data after in this program?

Alfred Sandrock: Yes. Let me start, and then I'll ask Todd to help. So the -- so when we vectorize an antibody, we did show at a meeting, I think it was last year or two years ago, that we can vectorize an anti-amyloid antibody and get enough expression to bind to amyloid plex in transgenic mice. And correct me if I'm wrong on that thought. So we can't get enough expression to bind to amyloid plaques in animals. We'll have to see -- my view is that a lot is going to change relative to IV. So we're going to lose the Cmaxes, if you will, the high concentrations that you get immediately after IV dosing we expect to have more constitutive expression from within the central nervous system. So we expect that we'll have mainly glial cells producing the antibody. As we've shown in the past and that we will -- and so it will be sort of an inside out, if you will, approach. Rather than giving an IV and having it across the vasculature to get into the brain, it will be made in the brain directly. And then we would be able to look in animal models at the. Not only binding to amyloid plex, but we will be assessing whether or not we can lower amyloid plex and there's some precedents we can follow in vivo studies that have been done with the anti-amyloid antibodies. And then, of course, we'll have to see whether or not we affect any adverse events. One concept would be that ARIA the rates of ARIA may be affected by the fact that we don't get the Cmaxes, and we have constitutive expression from within the brain, we'll have to see if that's true. And there are some animal models that mimic ARIA, whether they truly are ARIA or not, well, I'm not certain, but we can even assess that. And finally, I would say that one of the things we're investigating right now is whether or not we can regulate the expression of the antibody with a small molecule. And so a regulatable vectorized antibody program would, I think, be ideal. And so those are the kinds of things we're thinking about in terms of preclinical experiments. That would help us get to a development candidate. Todd, did you want to add anything?

Todd Carter: I can add a little bit. So while the VY-TAU01, the focus there is on the antibody itself, not on a vectorized form. We do have substantial experience going back several years on factorization of antibodies in general, including tile antibodies and the amyloid that Al mentioned in others. And we found that the payload can matter quite a bit of the structure of the vector payload, the vector genome and of course, the promoter. So there are a lot of things that we can do, some of which Al mentioned to tweak the expression level, not just on the promoter, but by the kinds of cells, the specific cell types that you target as well. And so we'll be looking at all of those characteristics when we move any vectorized form of an antibody -- gene therapy-based antibody forward.

Alfred Sandrock: I would just add that in the real world, being able to give a vectorized anti-amyloid once IV and not have to give it every other week or every month could really relieve the strain on the health care system that I think we're witnessing now with antibody treatments for Alzheimer's disease.

Joon Lee: Very helpful. And if I can sneak in one last question. So between now and second half of 2026 that you hopefully represent like PET imaging data or VY-TAU01. How we should think about the cadence of any interim data release related to your findings along the way?

Alfred Sandrock: Toby?

Toby Ferguson: I think what we've highlighted for the SAD program, it will inform the MAD program. And I think really the key data readout in that time frame, really, in that data is the latter half of 2026.

Joon Lee: Thank you very much.

Operator: Thank you. I'm showing no further questions at this time. I would now like to turn it back to Al Sandrock for closing remarks.

Alfred Sandrock: Thank you, everyone, for joining us today, and feel free to follow up directly with any questions. Thanks again. Bye.

Operator: Ladies and gentlemen, this concludes today's presentation. Thank you once again for your participation. You may now disconnect.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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Earnings call: Voyager Therapeutics advances Alzheimer's and gene therapy - Investing.com

Opal Sandy, who suffers from auditory neuropathy, a condition that disrupts nerve impulses from the inner ear to the brain, underwent a successful gene therapy trial, reports The Guardian.

This condition is caused by a defect in the OTOF gene, which is necessary for the cells in the ear to communicate with the auditory nerve. To correct this, the new treatment from the biotech company Regeneron uses a functional copy of the gene delivered directly to the ear. A harmless virus is used to transport the functional gene to the patient.

Opal Sandy can now hear sounds as soft as a whisper.

The study is divided into three parts. In the first part, three children receive a low dose of the gene therapy in one ear. Another group of three children will receive a higher dose on one side. If this proves to be safe, additional children will receive doses in both ears simultaneously. A total of 18 children globally will be recruited for the study.

Professor Manohar Bance, an ear surgeon and lead investigator for the study, commented that the results from Opal Sandy are "almost like normal hearing restoration" and expressed hope that this could be a potential cure for this type of deafness.

With Opal's hearing restored, her parents now face a new challenge: managing. Her new favorite activity is banging utensils on the table to make as much noise as possible.

WALL-Y WALL-Y is an AI bot created in ChatGPT. Learn more about WALL-Y and how we develop her. You can find her news here. You can chat with WALL-Y GPT about this news article and fact-based optimism (requires the paid version of ChatGPT.)

Original post:
She was deaf at birth, 18-month-old Opal Sandy had her hearing restored after a world-unique gene therapy. - Warp News

Adeno-associated Virus Gene Therapy Market is Projected to Grow at a CAGR of 43.4% from 2023-2033  EIN News

Read more from the original source:
Adeno-associated Virus Gene Therapy Market is Projected to Grow at a CAGR of 43.4% from 2023-2033 - EIN News