Category Archives: Gene therapy

Robin Alderman faces an agonizing reality: Gene therapy might cure her son Camdens rare, inherited immune deficiency. But its not available to him.

In 2022, London-based Orchard Therapeutics stopped investing in an experimental treatment for the condition, Wiskott-Aldrich syndrome. And there are no gene therapy studies he can join.

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Excerpt from:
Gene therapy may cure rare diseases. But drugmakers have few incentives, leaving families desperate - goskagit.com

The US Food and Drug Administration (FDA) has granted expanded approval to Sarepta Therapeutics Duchenne muscular dystrophy (DMD) gene therapy to treat patients aged four years and older with a confirmed mutation in the DMD gene.

Elevidys (delandistrogene moxeparvovec-rokl) was first authorised under the US regulators accelerated approval pathway in June last year for use in ambulatory patients aged between four and five years.

The one-time treatment has now been granted traditional approval to treat ambulatory patients aged four years and over, and accelerated approval to treat non-ambulatory patients in the same age group.

Estimated to affect one in every 3,500 male births worldwide, DMD is a severe genetic condition caused by a change or mutation in the gene that encodes instructions for dystrophin, which is required to strengthen and protect muscles.

Symptoms are usually first seen in early childhood and worsen over time, affecting patients ability to walk as well as their heart and respiratory muscles.

Administered as a single intravenous dose, Sareptas therapy is designed to address the underlying cause of DMD by delivering a gene into the body that leads to the production of Elevidys micro-dystrophin, a shortened protein that contains selected domains of the dystrophin protein present in normal muscle cells.

The FDAs latest decision on the therapy was supported by results from two double-blind, placebo-controlled studies and two open-label studies, which enrolled a total of 218 male patients with a confirmed disease-causing mutation in the DMD gene.

Doug Ingram, Sareptas president and chief executive officer, described the label expansion as a defining moment for the Duchenne community.

Sharing a similar sentiment, Jerry Mendell, co-inventor of Elevidys and senior advisor of medical affairs at Sarepta, said: The initial approval of Elevidys was a significant milestone, and the expanded indication means clinicians now have a treatment option for the great majority of boys and young men living with Duchenne.

This expansion speaks to the success of the science, the evidence and the improvements in the trajectory of the disease we have seen to date across studies.

Sarepta is responsible for regulatory approval, commercialisation and the manufacturing of Elevidys in the US, while Roche is responsible for regulatory approvals and bringing the therapy to patients across the rest of the world.

See the article here:
FDA expands indication for Sareptas Duchenne muscular dystrophy gene therapy Elevidys - PMLiVE

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Gene Therapy-Rare Disease | National | wataugademocrat.com - Watauga Democrat

Shares of Sarepta (SRPT Quick QuoteSRPT - Free Report) rose nearly 34% in after-market trading on Thursday after it announced that the FDA approved the expanded use of its Duchenne muscular dystrophy (DMD) gene therapy Elevidys.

Elevidys is now approved to treat all DMD patients aged four years and older. While the FDA granted traditional approval for the therapy to treat ambulatory DMD patients (those who can still walk), it has granted accelerated approval for non-ambulatory patients.

Sareptas gene therapy was initially approved by the FDA last year under the accelerated pathway to treat ambulatory pediatric patients aged between four and five years with DMD. Following the FDA nod, Elevidys was the first-ever approved gene therapy for DMD.

The label expansion is mainly supported by data from the phase III EMBARK study, announced last October. Though the study failed to achieve its primary endpoint, it achieved statistical significance on all pre-specified key secondary endpoints, indicating that treatment with Elevidys modifies the course of DMD indication.

Management will still need to conduct a confirmatory study to convert the accelerated approval for non-ambulatory DMD patients to a full one. Sarepta is currently conducting the phase III ENVISION study to evaluate the safety and efficacy of gene therapy in non-ambulatory and ambulatory DMD patients. This study also satisfies the regulatory requirements for Elevidys approval outside the United States.

Elevidys is the only one-shot gene therapy for DMD in the United States. A progressive and degenerative disorder, DMD leads to weakness and wasting away of the bodys muscles. Despite being initially approved with a confined label, Sarepta recorded revenues of more than $200 million from Elevidys sales last year, which is an encouraging figure for a therapy that was commercially launched in the second half of 2023. This uptick in share price, following the label expansion announcement, is likely attributed to Elevidys blockbuster potential.

Year to date, Sareptas shares have inched up 28.1% against the industrys 7.9% fall.

Image Source: Zacks Investment Research

Sarepta developed Elevidys in collaboration with Roche (RHHBY Quick QuoteRHHBY - Free Report) . Sarepta and Roche entered into a licensing agreement in 2019 to develop and commercialize Elevidys. Per the agreement, Sarepta is responsible for marketing the therapy in the United States, while Roche is responsible for marketing the gene therapy outside the country. Sarepta is also eligible to receive collaboration revenues on the ex-U.S. sales made by Roche.

Sarepta is currently the market leader in DMD treatment. Apart from Elevidys, the company has three other therapies in its commercial portfolio targeting the DMD patient population, namely Exondys 51, Vyondys 53 and Amondys 45. Per management, these three drugs have the potential to address nearly a third of all patients with DMD in the United States.

However, competition in the DMD space is stiff as companies like Regenxbio (RGNX Quick QuoteRGNX - Free Report) and Solid Biosciences (SLDB Quick QuoteSLDB - Free Report) have been developing their respective gene therapy candidates for DMD.

Regenxbio is planning to hold an end-of-phase II (EOP2) meeting with the FDA early in the next quarter to discuss the study design of a pivotal late-stage study on RGX-202, its DMD gene therapy candidate. Based on the feedback, Regenxbio expects to start this pivotal study before 2024-end. If the outcome of this study is positive, it plans to seek accelerated approval for its therapy from the FDA.

Last year, the FDA cleared Solid Biosciences investigational new drug (IND) to start a phase I/II study on SGT-003 in pediatric patients with DMD. This study is expected to begin by the end of this month. SLDB expects to report initial data from this study by this years end. The FDA has granted fast-track, orphan drug and rare pediatric disease designations to Solid Biosciences gene therapy in DMD indication.

However, companies also have suffered major setbacks in the DMD space. Earlier this month, Pfizer (PFE Quick QuotePFE - Free Report) announced that a late-stage study evaluating its DMD gene therapy candidate failed to meet the primary endpoint and key secondary endpoints. Treatment with the Pfizer therapy failed to improve motor function among the DMD boys. Pfizer plans to share more detailed results from the study at an upcoming medical meeting.

Sarepta sports a Zacks Rank #3 (Hold). You can see the complete list of todays Zacks #1 Rank (Strong Buy) stocks here.

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FDA Expands Sarepta's (SRPT) DMD Gene Therapy Label - Zacks Investment Research

Pictured: A boy in a wheelchair over a muscle texture background/Taylor Tieden forBioSpace

In a much-anticipated regulatory decision on Thursday, the FDA significantly expanded the label and use of Elevidys, Sarepta Therapeutics gene therapy for Duchenne muscular dystrophy (DMD). Most exciting, perhaps, is the expansion of Elevidys label to include older individuals as well as non-ambulatory ones, broadening the spectrum of patients eligible for this breakthrough therapy. While Elevidys was previously approved under accelerated approval for ambulatory individuals 4 through 5 years with a confirmed mutation in the DMD gene, Thursday's expanded approval includes ambulatory and non-ambulatory patients 4 years of age and older with a confirmed mutation in the DMD gene.

It was a year ago that the regulator granted accelerated approval for Elevidys as the first FDA-approved gene therapy for DMDdespite falling short of its primary efficacy endpoint in two studies. In making its decision, the FDA on Thursday said it considered the totality of the evidence, including the potential risks associated with the product, the life-threatening and debilitating nature of the disease and the urgent unmet medical need.

Under its previous label, Elevidys was limited to only about 3% of the total DMD population, according to Sarepta CEO Doug Ingram. However, BMO Capital Markets analyst Kostas Biliouris, in a note to investors, said that with Thursdays approval, the FDA has effectively opened Elevidys label to more than 90% of patients in the U.S.

Elevidys is now approved to treat a population that includes ~13,000 DMD patients (~90% of total prevalence), creating a significant commercial opportunity for [Sarepta], Biliouris wrote.

William Blair analyst Tim Lugo agreed, writing in an investor note that Thursdays FDA approval is the best-case scenario for Sarepta since many patients below age 4 are not yet diagnosed but will age into the label, opening the DMD market to all current and future patients who will be eligible for treatment. Lugo also highlighted the fact that Sarepta has an ongoing Phase II study for boys under the age of 4.

Its also worth mentioning that Elevidysdeveloped and commercialized in partnership with Rochehas a $3.2 million per patient price tag, making it one of the worlds most expensive medicines. Data analysis firm GlobalData said it expects Sarepta to pocket $5.7 billion in Elevidys sales by 2029.

Biliouris said in his investors note that Sarepta will have a monopoly in DMD for the next ~4+years. That 4+ years of monopoly is thanks in part to the stumble by Pfizers experimental DMD gene therapy, which was found not to improve motor function versus placebo in a Phase III trial last week.

Indeed, there is only one other company with a DMD candidate in Phase III: Capricor Therapeutics cell therapy CAP-1002. The company expects topline data from the ongoing trial by the end of this year. Other DMD treatments are all in earlier stages of development.

Jeff Chamberlain, president of the American Society for Gene and Cell Therapy and the McCaw Chair in Muscular Dystrophy at the University of Washington School of Medicine, told BioSpace last month that Sarepta has done a fantastic job on Elevidys, with some children treated with the gene therapy doing tremendously well, while others he said are not doing quite as well.

In the coming years, it is critical to overcome the challenges with the first generation of micro-dystrophin gene therapy products, according to Chamberlain. Just because we have an approved therapy doesnt mean you can stop and go home, Chamberlain said. We want something thats going to be more effective; thats going to work on all patients, not just some of them.

Greg Slabodkin is the news editor at BioSpace. You can reach him atgreg.slabodkin@biospace.com. Follow him onLinkedIn.

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Expanded Approval of Sareptas Elevidys Is Progress, But More Needed for DMD Patients - BioSpace

Ascidian to receive $42 million in initial payment, and up to $1.8 billion in research, clinical and commercial milestone payments, as well as commercial royalties

Per-target agreement enables Ascidian to pursue additional internal and collaborative programs within neurology and other therapeutic areas

Combines RNA Exon Editors with next generation CNS delivery capabilities of Roche to develop novel medicines for difficult to treat neurological diseases

BOSTON, June 18, 2024 /PRNewswire/ --Ascidian Therapeutics, a biotechnology company seeking to treat human diseases by rewriting RNA, today announced a research collaboration and licensing agreement with Roche (SIX: RO, ROG;OTCQX: RHHBY) for the discovery and development of RNA exon editing therapeutics targeting neurological diseases.

Ascidian's RNA exon editing platform is designed to advance the therapeutic possibilities of RNA medicine and treat diseases not addressed by today's gene editing technologies. The company designs and develops RNA exon editing therapeutics that edit RNA exons at the kilobase scale.

Under the agreement, Ascidian will provide Roche exclusive, target-specific rights to Ascidian's RNA exon editing technology for undisclosed neurological targets. Ascidian will conduct discovery and certain preclinical activities in collaboration with Roche, and Roche will be responsible for certain preclinical activities, and further clinical development, manufacturing, and commercialization. Ascidian will receive an initial payment of $42 million and is eligible to receive up to $1.8 billion in research, clinical, and commercial milestone payments, as well as royalties on commercial sales worldwide. Based on the terms of the agreement, Ascidian is free to develop programs against other neurological targets internally or with other collaborators.

"Roche is known and respected worldwide for their expertise in complex neurological diseases, and I am proud of the scientific rigor and quality of the work done at Ascidian that has led to this partnership," saidMichael Ehlers,M.D., Ph.D., President and Chief Executive Officer of Ascidian Therapeutics. "The potential of treating disease by large-scale exon editing of RNA is vast. We look forward to working with the Roche team to develop first-in-class RNA exon editing medicines for multiple neurological diseases, with a mission and passion to relieve suffering and improve lives."

"Our partnership with Ascidian is an opportunity to harness advanced RNA exon editing technology, which has the potential to deliver transformative one-time therapeutics by editing multiple whole exons at the RNA level with a single treatment," said James Sabry, M.D., Ph.D., Global Head of Pharma Partnering at Roche.

Ascidian's platform enables targeting of large genes and genes with high mutational variance while maintaining native gene expression patterns and levels. By rewriting RNA, Ascidian's exon editing technology is designed to provide the durability of gene therapy, while sharply reducing risks associated with direct DNA editing and gene replacement.

About Ascidian Therapeutics

Ascidian Therapeutics, an ATP company, is redefining the treatment of disease by rewriting RNA. By editing exons at the RNA level, Ascidian therapies enable precise post-transcriptional editing of genes, resulting in full-length, functional proteins at the right levels, in the right cells, at the right time. With discovery, preclinical, and clinical programs in retinal, neurological, neuromuscular, and genetically defined diseases, Ascidian's approach has the potential to treat patients with one dose of an RNA exon editor, opening new therapeutic possibilities for patients and their families who are seeking breakthroughs. Earlier this year, Ascidian announced U.S. FDA IND clearance for the first-ever RNA exon editing candidate, ACDN-01, which targets Stargardt disease and other ABCA4 retinopathies. Ascidian is currently executing the Phase 1/2 STELLAR clinical trial to evaluate the safety and efficacy of ACDN-01. For more information, visit http://www.ascidian.com.

SOURCE Ascidian Therapeutics

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Ascidian Therapeutics Enters Collaboration with Roche for Discovery and Development of RNA Exon Editing ... - PR Newswire

Inspiration sparked by shaving

Stephan co-founded Tidal Therapeutics to help commercialize his immune-cell programming nanoparticles (acquired by Sanofi in 2022). His lab needed a new direction, and inspiration struck one morning as he watched his freshly sprayed shaving foam expand in his palm.

I thought, Let's explore foam, Stephan said. Maybe we can make a formulation of foam that is not like the foam in in our shaving foam, but something that is biocompatible, to deliver therapeutics.

The froth had properties that would be attractive in a drug-delivery vehicle. Its volume started small, but puffed up. The foam stayed where it was sprayed, and didnt slide away. These characteristics could help get a therapeutic into contact with more critical cells while also ensuring that it didnt slip away.

Trying new substances or approaches that come from things in everyday life that you wouldn't necessarily associate in medical applications is sometimes a really interesting way to drive down costs and deliver drugs more easily, Fitzgerald said. But it was definitely a little bit out of my wheelhouse.

Foam-as-medical-delivery method isnt without precedent, Stephan noted. Foam-based delivery already enhances certain applications like delivery of hemorrhoid medication and intra-uterine imaging.

But could foam enhance gene therapy?

To create a bio-compatible foam, Stephan and his team initially took inspiration from the food industry.

Cocktails, ice cream, yogurt: they know how to make things foamy, he said.

Stephan Lab members, including staff scientist Sirka Stephan, PhD, started experimenting with ingredients available from the pantry store, he said.

Importantly, these materials are dead cheap, Stephan said. Theyre available for pennies. Theyre manufactured at large scale, and because theyre already used for pharmaceutical applications like coating tablets theyre available pharmaceutical-grade.

The scientists formulated a solution of methylcellulose (a food binder) and xanthan gum (a food thickener) that, when aerated using two lab syringes, bubbles into an easy-to-apply froth.

But did it have the potential to improve gene therapy?

We started with a lot of hypotheses in terms of, could foam potentially concentrate our gene therapy, keep it more localized, and help it stay in the tissue where we wanted to adhere? Fitzgerald said. My job was to do the experiments to prove the hypotheses.

Foam has certain properties that make it an attractive drug-delivery vehicle. Its more than tightly packed air bubbles: In a foam, the bubbles are separated and surrounded by incredibly thin layers of continuous liquid, called lamellae. Active ingredients become highly concentrated in these lamellae, which allows foam to deliver highly concentrated doses of medicine to large areas, even if the total dose is small.

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Taking a frothy risk to advance gene therapy - Fred Hutchinson Cancer Center

SEATTLE It is a virus that's been ailing humans for millions of years, but to this day, there has been no absolute cure.

Virologistsat Fred Hutchinson Cancer Center in Seattle believe they're on their way to finding one through gene therapy after pre-clinical models showed it reduced up to 90 percent of the viral infection in tests.

"We're working on trying to develop new, gene therapy-based cures. Not just to treat these viruses but actually to cure them," saidDr. Keith Jerome, a professor in the Vaccine and Infectious Disease Division at Fred Hutch and one of the researchers of the study.

Jerome's research specialty is on persistent viruses like HIV, Hepatitis B, and herpes simplex virus.

"We're working on trying to develop new, gene therapy-based cures. Not just to treat these viruses but actually to cure them," Jerome said.

Herpes simplex virus 1, or HSV 1, mostly shows up as painful blisters around the mouth, commonly referred to as "cold sores," but it can also infect the genital region, according to Jerome.

"These are really common. I mean over half the people in the world have one or the other or both, so they're very common infections," Jerome said.

According to the latest available data from theWorld Health Organization, 3.7 billion people under age 50 worldwide have HSV 1. An estimated 491 million people between age 15 and 49, have herpes simplex 2, or HSV 2, the virus that causes genital herpes infections.

While there are existing and effective anti-viral herpes treatments available today, Jerome said they don't solve the problem. He said the herpes virus, in particular, buries itself deep into nerve cells and can go dormant.

"The viruses hang out there in the form that stays for life," Jerome said.

So while anti-virals are at-the-moment treatments, there has not yet been an outright cure.

"That long-lived form in the nerves is not even touched. So it can always come back and always start new episodes," Jerome said.

Jerome and his team believe they're on their way to finding a cure by way of gene therapy to get rid of herpes infection for good.

Their findings,publishedin the journal Nature Communications in May, showed they were able to eliminate at least 90 percent of the virus from nerves in animal testing models.

"What we're doing with our gene therapy is actually sending a protein into those nerve cells that actually looks for the virus and basically snips it just like scissors, cuts it in a couple of places, and the body recognizes, it suddenly can see that, and gets rid of it.

The gene therapy also significantly lowered the frequency and amount of viral shedding in animal testing and worked as effectively, if not more, for genital HSV 1 infections, according to Jerome.

"And that's the late, long-lived form we're actually getting rid of," Jerome said.

It's a long sought-after cure, not only because of its prevalence but the stress, even stigma, it causes.

"For some people, it is not a big deal. For other people, it's an enormous deal. And that's each person's lived experience. What we want to do is offer a tool that says if this really matters to you, we can help make your life better," Jerome said.

While the pre-clinical phase showed promising results, the gene therapy has yet to enter clinical trials and is not available as a treatment now.

If approved, Jerome sees the treatment administered as an injection.

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Promising new results for potential herpes cure: HealthLink - KING5.com

The progress of advanced therapeutics in recent years, especially in oncology, have been so promising many are starting to use the term cure when talking about some types of cancer. Despite complex manufacturing processes, multiple cell and gene therapies are providing front-line treatment options, with others continuing to move closer to front-line.

In many cases, producing a cell therapy involves modifying primary immune cells to recognize, seek out, and attack specific cancer cells, which can co-opt immunosuppressive mechanisms to evade the bodys immune defenses.

As scientific understanding develops in parallel with technological advances, the next generation of cell and gene therapies addressing a wide array of conditions and diseases are expected to be developed and commercially approved. Oncology is the dominant therapy area, which is widely expected to continue in the years ahead. Within the next decade, treatment options are also understood to be possible for central nervous system conditions and immunology patients.

Recent breakthroughs in cell and gene therapy build on research knowledge and expertise that span decades. The first chimeric antigen receptor (CAR) T-cell therapies were tested for oncology in the late 1990s and early 2000s. These early CAR-T cell therapies did not show the proper durability for eliminating cancer, so considerable efforts were focused on increasing that durability. The turning point came in 2012, when several manuscripts were published describing the first CAR-T patients who were in remission because of the therapy. This was followed by what is now seen as a pivotal moment in the field: the treatment of the first pediatric patient, Emily Whitehead.

After the 2012 milestones, the intensity of cell therapy development increased and culminated in a pivotal year, 2017, when the U.S. Food and Drug Administration (FDA) approved the first CAR-T therapy, Kymriah. Later in 2017, the European Medicines Agency (EMA) approved its first CAR-T therapy, Yescarta. Between 2017 and the end of 2022, a total of six therapy products were approved by the FDA.1

The growth of commercial cell therapies continues. In 2023 alone, there were eight U.S. cell therapy commercial approvals by FDA. Depending on progress in 2024, the U.S. may see more than ten products approved.

The advanced therapy preclinical and clinical pipeline also continues to show promising growth. There are approximately 1,800 active clinical trials worldwide, with 60% having at least one clinical site in the U.S.2 This seems to suggest the field will continue to see commercial approvals. In cell therapy, multiple products address blood cancers, including multiple myeloma, leukemias, and lymphomas. The field hit another crucial milestone in February 2024 when the FDA approved the first tumor-infiltrating lymphocyte (TIL) therapy for solid tumors.2

This first-line TIL therapy has been designed to treat melanoma. As with CAR T-cell therapies, Iovance Biotherapeutics Amtagvi involves taking cells from the patients body and enhancing them externally before re-administering them to the patient, where they then attack tumor cells.

Weve already proven that CAR-T works in leukemia and lymphoma, now were starting to prove that new technologies harnessing neoantigens, TIL, CAR-T, and others can now be applied to the next therapeutic hurdle of solid tumors, explains Dr. Phil Vanek, chief technology officer at Gamma Biosciences.

There remains a supply and demand imbalance in the advanced therapies space, specifically in cell therapies that target cancer. A survey of 17 clinical centers in the U.S. led by Dr. Yin Lin at the Mayo Clinic in Minnesota, the median waiting time for patients to receive a commercial CAR-T therapy was six months.

Survey results revealed in 2022 that only 25% of patients received the commercial CAR-T therapy for which they qualified, and another 25% of patients joined a different CAR-T clinical trial because of their inability to access their preferred commercial CAR-T therapy.3 The remaining 50% of patients either entered an alternative type of clinical trial or moved into a hospice and died.3

Commercial cell therapy costs run into the hundreds of thousands of dollars, and this presents a potential barrier to adequate patient access. Matthew Hewitt, CTO for CGT and Biologics at Charles River Laboratories (CRL), emphasizes the need to prioritize

identification and commercialization of promising new therapies. Its important for these therapeutic developers to work with partners and help them think through important considerations about manufacturing, costs, and regulatory approval.

While everyone agrees we need to address costs, first we need commercial therapies then we can continue to work on improving them from a cost perspective, he says. If you look at history, the pharma space has a pretty good track record of innovating our way out of problems.

There are a lot of technologies in development and coming to the market which can help bridge that gap, adds Hewitt.

Development timelines for cell therapies have shortened notably in recent years. In previous generations, it was common to have timelines of ten to 12 years for a biologic product to progress from discovery to clinical trials and commercial authorization. But timeframes for CAR-T therapies are substantially shorter.

The first CAR-T was approved in just a few short years from the first therapeutic trials, all the way through commercialization. That shortened our cycle time for development and were still working to improve it, says Vanek.

Despite the pharma industrys historic reputation for being slow-moving, Vanek explains that the rapid pace of development of CAR-T therapies forced the industry to adapt processes and manufacturing technologies. One of the biggest challenges was production being heavily reliant on tools and technologies built for the bioprocessing industry.

The industry was building the plane while we were flying it. We were scrambling to keep up. But that was a good thing, adds Vanek. Clinical success must be at the heart of everything. We can anticipate what might be successful. But when you see a binary clinical success, like a CAR-T therapy, where people go into remission and remain that way, that was a real wake-up call to say: This therapeutic modality is real, its here, its now. We must adapt how we make these things to make them widely available.

To meet demands for pace and scale, life sciences and tech companies have built equipment and analytical technologies that combine manufacturing and analysis to drive greater efficiencies in development and production. As a direct result, the industry has learned how to consistently make many different types of cell and gene therapies.

Weve taken out a lot of the variability in manufacturing to make these therapies much more robustly and reproducibly, says Vanek. Weve also reduced a lot of the therapeutic and regulatory risk by removing animal component materials and adding well-characterized raw materials. Weve worked out the supply chain in terms of receipt of materials, freezing cells and thawing, and keeping cells throughout the manufacturing workflow both viable and healthy.

In terms of tracking cells for autologous patients, were using a much more significant chain of custody and chain of identity capability to strengthen the entire supply chain of cell and gene therapy manufacturing.

As we reduce the cost of manufacturing, there will be pressure in the industry to make these therapies more affordable and more widely available.

There is much optimism within the industry about the future, with thousands more cell and gene therapies undergoing clinical trials. Despite many remaining challenges, the industry clearly wants to develop the necessary solutions.

We know autologous works, explains Hewitt. So, we should also be pushing forward better autologous development of therapeutics, as well as better equipment to allow us to manufacture these therapies in a larger scale more cheaply without compromising safety and quality.

Built on an understanding of oncology, cell and gene therapies could one day provide effective treatments for a range of infectious diseases and conditions such as diabetes, as well as improve the health of neurological patients. There is also much excitement surrounding therapies in the autoimmune space, as well as the ongoing discoveries of new cell types that create further opportunities.

Looking at the autoimmune space, were seeing some really good data there, adds Hewitt. IPSCs (induced pluripotent stem cells) should also be in the conversation. These are cells with the ability to become any cell type with the right stimuli. They have applications both within and outside of oncology, and more going into regenerative medicine.

Also, on the allogeneic side, most programs are using donors to supply their starting material. Campaigns to find good donors can be very expensive and time-consuming. By moving over to an IPSC cell bank, you could eliminate that and, in principle, have an evergreen supply of starting material.

Regulatory approval pathways could also be streamlined, with the innermost components of an established therapy fine-tuned to meet the needs of specific patients. All these regulatory and manufacturing concepts start to evolve into almost push-button therapeutic manufacturing production methods, producing customized products for end users, adds Vanek. I think thats doable in our lifetime.

The more therapy products that undergo clinical trials and enter the market, the more data will be available to refine treatments and advance therapies further.

This convergence of readily available biological data and our ability to mine big datasets really unlock a lot of potential and value, says Vanek.

Due to the increases in data, uses of artificial intelligence (AI) are widely predicted to increase. Notably, Charles River has been testing out AI in certain processes, having developed a platform called Logica in partnership with Valo Health for drug discovery and design, with several licensing deals announced. The Logica platform has primarily been used for small-molecule development, with other uses across the business being considered.

For preclinical development, and even in a use case like pathology, this could be very useful to help lead developments more efficiently. We have a goal at Charles River to cut up to 12 months off the overall therapeutic development timeline. That wouldnt be in one particular area, it would be across the different parts of therapeutic development, making it more efficient, says Hewitt. There are a lot of use cases we can apply AI to in process and analytical development to ensure that were applying robust standards that give us the best, most stable product process and analytical suite.

The combination of enhanced scientific understanding, growing volumes of data, and advances in manufacturing technologies are all pointing to a future market where the full potential of cell and gene therapy is unlocked across an extensive range of medical treatments for greater numbers of patients.

References:

1. Chen YJ, Abila B, Mostafa Kamel Y. CAR-T: What is next? Cancers (Basel). 2023;15(3):663. doi: 10.3390/cancers15030663.

2. GlobalData. lovances Amtagvi for melanoma treatment grains FDA approval. PharmaceuticalTechnology.com. 19 February 2024. https://www.pharmaceutical-technology.com/news/iovance-amtagvi-melanoma-fda/?cf-view

3. Nelson R. Patients waiting months for Last Chance CAR T-Cell Therapy Medscape.com. 14 July 2022. https://www.medscape.com/viewarticle/977179?form=fpf#vp_1:~:text=The%20team%20contacted,hospice%2C%20or%20died.

AMTAGVI and Iovance are either registered trademarks or trademarks of Iovance Biotherapeutics, Inc. in the United States and/or other countries. Kymriah is either a registered trademark or a trademark of Novartis in the United States and/or other countries. Yescarta is either a registered trademark or a trademark of Kite Pharma, inc. in the United States and/or other countries. Logica and Charles River are either registered trademarks or trademarks of Charles River Laboratories, Inc. in the U.S. and/or other countries. Valo Health is either a registered trademark or a trademark of Valo Health, LLC in the U.S. and/or other countries.

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How cell and gene therapy are evolving - Clinical Trials Arena

Ultragenyx said Thursday afternoon that its gene therapy for a rare liver ailment, glycogen disease type 1A, succeeded in a Phase 3 trial, setting up a potential approval.

Patients with GSD1a, as the condition is often known, have a genetic mutation that prevents them from adequately maintaining blood sugar levels. Once considered fatal, GSD1a can now be controlled with regular doses of cornstarch. But if patients miss a dose, there can be significant, even life-threatening, complications.

In the 49-person, randomized study, patients who received the Ultragenyx drug were able to take 41% less cornstarch after 48 weeks than they did at the start. Patients on placebo took only 10% less. The difference was statistically significant. Five patients were not included in the analysis, as three dropped out and the company didnt have 48-week data from two.

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Ultragenyx gene therapy for rare liver ailment succeeds in late-stage trial - STAT