Category Archives: Gene therapy

French biotech company WhiteLab Genomics has raised $10 million in funding for an AI platform designed to aid the discovery and development of genomic therapies.

Founded out of Paris in 2019, recent Y Combinator (YC) graduate WhiteLab Genomics provides gene and cell therapy companies with predictive software simulations to expedite the design of gene and cell therapies. Gene therapy, for the uninitiated, is an emerging treatment that involves replacing missing or defective genes in cells to correct genetic disorders, while cell therapy is about altering a cell or sets of cells to trigger an effect throughout the body.

Thousands of diseases, including cystic fibrosis, Parkinsons and Alzheimers stem from flaws in an individuals DNA, and emerging research in gene and cell therapies may eventually treat such conditions at their source, supplanting the need for drugs or surgery.

However, such therapies are typically costly to develop with no guarantee that theyll work. Current methodologies in developing new gene and cell therapies typically involves a trial-and-error approach, according to WhiteLab Genomics CEO and co-founder David Del Bourgo, whereby scientists make a scientific hypothesis and test it in a lab: if successful, it progresses to the next stage, but if its unsuccessful, they go back to square one with a different hypothesis. And this is where WhiteLab Genomics enters the fray, with a computational approach that meshes machine learning and deep learning techniques to process multiple scientific hypotheses at once, looking at different genetic variants to predict the best molecular design for the therapy based on the objectives.

Gene and cell therapies still suffer from poor efficacy, immunological secondary effects and very high development cost, Del Bourgo explained to TechCrunch. We provide customers with exhaustive predictive models combining genetics and computational biology in order to design and select the top candidates to be tested in the lab.

WhiteLab Genomics founders: CEO David Del Bourgo and Chief Science Officer Julien Cottineau. Image Credits: WhiteLab Genomics

As with just about any AI models, WhiteLab Genomics uses myriad datasets to train its algorithms spanning genomic, RNA, oncologic and protein repositories, in addition to an exhaustive set of documentation such as public scientific and clinical research data.

We also partner with specialized institutions to enrich the models with additional non-public data, Del Bourgo added. We train and validate our algorithms on well-characterized datasets that are interconnected within our Knowledge Graph Database. These included public databases, proprietary data and datasets from our partners. It predicts biological features from new genetic sequences to build new therapeutic vectors and generates new therapeutic constructs.

In terms of the kinds of treatments WhiteLab Genomics is helping to develop, Del Bourgo said that the company is currently working on projects including DNA-based therapies for metabolic conditions such as lysosomal diseases, as well as cell stem therapies for blood diseases such as sickle cell disease and immuno-gene therapies to treat cancers.

Elsewhere, anumber of companies are using AI to aid drug discovery, such as BenevolentAI, which has raised sizeable investments from the VC world, while theres TechCrunchs Startup Battlefield winner Cellino, which recently raised $80 million to develop cell therapies using AI. WhiteLab Genomics is similar, but its working with both gene and cell therapy companies.

Its also worth noting that WhiteLab Genomics represents a growing roster of French startups to successfully infiltrate the YC ranks. Of the 40 European companies in WhiteLab Genomics Winter 22 cohort, five were French, a figure that rose to eight of 34 for the Summer 22 tranche the second highest number from the whole of Europe.

With $10 million in the bank from French investor Omnes Capital and biopharmaceutical heavyweight Debiopharm, WhiteLab Genomics is now well-financed to build out its platform and double-down on its partnerships with the scientific realm, which so far has included notable collaborations with the French National Institute of Health and Medical Research (Inserm) and Genethon Laboratories, among other pharmaceutical and biotech companies.

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How WhiteLab Genomics is using AI to aid gene and cell therapy development - TechCrunch

TumorGen and PhenoVista Biosciences partnered toexplore metastatic cancer cell clustersto validate technology targeting these clusters and prevent cancer metastasis, which causes 90% of cancer-related deaths.

Using PhenoVistas analytical imaging technology, the two companies, whopartnered last year, reported TumorGens micro-fluidic technology platform was able to detect multiple metastatic clusters from several lung cancer patients. The platform showed tremendous sensitivity and specificity, which proved to be more effective than previous efforts to collect and characterize the clusters.

The clusters contained cancerous and non-cancerous cells, James Evans, CEO of PhenoVista, noted in the announcement. The companies announced the advancements could lead to improved therapeutic options for these patients.

The study, which the National Cancer Institute supported, notes that these metastasized clusters can be captured. This will allow for multiple analyses that could identify weaknesses and potential therapeutic approaches, which TumorGen dubbed cluster busters. This therapeutic could then be able to target the cells and prevent metastasis.

Jeffrey K. Allen, founder and president of TumorGen, noted in the companys announcement that there is a tremendous unmet medical need for anti-metastatic therapies that can prevent tumors from forming across the body. He said the companys platform is able to identify where these metastatic cancer cell clusters are vulnerable, enabling the development of those needed therapeutics.

He told BioSpace the company is energized to pursue therapeutic targets that can lead to a whole new class of anti-metastatic drugs that will save lives.

Our next steps will be to expand our efforts beyond lung cancer to other solid tumor types, all of which have been shown to release circulating metastatic cancer cell clusters, he said. We have also begun discussions with a biopharmaceutical company about beginning to use our technology to characterize MCCCs, confirm biomarkers of interest, and evaluate therapeutic targets within their pipeline.

When it comes to looking for a partner, Allen said TumorGen hopes to pair up with a company that is looking to differentiatethemselves in the oncology space by focusing on stopping metastatic disease before it spreads.

Allen said that the majority of cancer therapies remain focused on the primary tumor, while the different mechanismscontrolling metastasis remain unaddressed. Since MCCCs are often heterogeneousand may contain immune cells that protect them from immune surveillance, Allen said they hope to find a partner interested in investigating targeting more than just the cancer cells within the cluster.

Gene Therapy Developed for Rare Blindness Disease

Researchers from theNational Institutes of Health havedeveloped a potential new gene therapy approachto Leber congenital amaurosis (LCA), a disease that causes blindness in children.

A research team from the National Eye Institute, a division of the NIH, revealed that mutations cause a type of LCA in the NPHP5 gene, which leads to defects in the primary cilium found in cells throughout the body,Technology Networksreported. A deficiency in NPHP5 can cause blindness in children. However, the team explained that deficiency could lead to kidney disease in severe cases.

Defects in 25 different genes can cause LCA. The FDA has approved Spark Therapeutics gene therapy Luxturna for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy, a form of LCA. That leaves other forms of this disease without treatment.

Using stem cells collected from two patients with NPHP5 deficiency, the NIH team generated retinal organoids, which possess functional features of the natural retina. A deficiency of NPHP5 was found in the retinal organoids, along with lower levels of the protein CEP-290, which works alongside NPHP5.

Using an adeno-associated viral vector that contained a functional version of NPHP5, the NIH researchers were able to target these extracted stem cells and demonstrate a significant restoration of opsin protein concentrated in the proper location in outer segments. The NIH research warrants further study and suggests a functional protein could prevent blindness in these children.

Further research is warranted, particularly in more severe forms of the disease.

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Cluster-busters against Cancer and a Potential New Gene Therapy for LCA - BioSpace

Wilmington, Delaware, United States, Transparency Market Research Inc. CAR T cell therapy is considered as an extremely effective strategy for the treatment of patients experiencing blood malignant growth and has effectively experienced other methods of disease treatment like chemotherapy. The effectiveness of the cycle in treating disease is probably going to fuel growth of the global CAR-T cell therapy market in the years to come.

Cancer is one of the main sources of fatalities around the world. Illusory antigen receptor or CAR-T cell therapy is used for fighting cancer cells, and includes re-designing of the white platelets of the cancer-affected patient. While CAR-T cell therapy, The T-cells of an individual are inferred, changed, and the put inside the body of the person where the restructured cells multiply themselves in number and afterward attack the cancer cells.

The development of the CAR-T cell therapy market worldwide is predicted to gather momentum from the increasing number of investors, designers, and analysts in the guide therapy. Rising frequencies of cancer across the globe together with the relentless technological advancement for dependable and effective cancer treatment is expected to support growth of the worldwide CAR-T cell therapy market. Furthermore, heavy investments on clinical research and development in cell therapy are estimated to boost the market for CAR-T cell therapy in the years to come.

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Global CAR-T cell therapy Market: Overview

Cancer is one of the leading causes of fatalities worldwide. Chimeric antigen receptor or CAR-T cell therapy comprises re-engineering of WBC or the white blood cells of the cancer-affected patient. This is fight off cancer cells. During the time of CAR-T cell therapy, the T-cells of a person are derived, reformed, and the put inside the body of a human being wherein the restructured cells multiply in number and then attack the cancer cells.

Currently, CAR T cell therapy is considered as an extremely effective procedure for the treatment of patients suffering from blood cancer and has already been through other methods of cancer treatment such as chemotherapy. The effectiveness of the process in treating cancer is likely to fuel growth of the global CAR-T cell therapy market in the years to come.

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Rising incidences of cancer across the globe together with the relentless technological advancement for reliable and effective cancer treatment is expected to favor growth of the global CAR-T cell therapy market over the period of projection, from 2019 to 2027. Furthermore, growing number of clinical studies in cell therapy is estimated boost the market over the period of forecast.

This report on the global CAR-T cell therapy market offers a closer look at the significant changes that took place in the industry of medical science. The developments are likely to shape the contours of the global CAR-T cell therapy market over the assessment tenure, from 2019 to 2027. The insights offered in the report are estimated to help the industry players and investors to make a correct assessment of the completive ecosystem and make strategies accordingly.

Global CAR-T cell therapy Market: Trends and Opportunities

Rising Geriatric Population with Weakening Immune System Boost Market Growth

The global CAR-T cell therapy market is primarily driven by increasing number of acquisitions, thanks to the augmented importance of cellular therapies. In addition to that, some of the acquisitions are done to facilitate more innovative research and development activities, which further boosts the global CAR-T cell therapy market in near future. A case in point is the leading role played by both Novartis AG and Gilead Sciences, Inc. following the approval of first therapies in pediatric Acute Lymphoblastic Leukemia and adult Diffuse Large B-cell Lymphoma or DLBCL.

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Scientists, investors, and developers agree unequivocally that solution to two major problems can offer long-term success in this field. One of them is the recognition of antigens except CD19 that could be efficaciously targeted with CAR-T cell therapy. The other problem lies in the indications of tumor, which lies beyond liquid cancers. The development of the global CAR-T cell therapy market is predicted to garner momentum from the rising interest of investors, developers, and researchers in the aid therapy.

Global CAR-T cell therapy Market: Regional Outlook

The five important regions in the global CAR-T cell therapy market include North America, Europe, the Middle East and Africa, Latin America, and Asia Pacific.

North America is estimated to take lead of the global CAR-T cell therapy market over the projection period. The dominance of the region is ascribed to the favorable policies of reimbursement, growing incidences of severe lymphoblastic leukemia, early availability of products of CAR-T cell therapy, and high per capita expenditure on healthcare. In addition, presence of a large number of clinical pipeline drugs that are expected to get approval for commercialization in near future is likely to fuel expansion of the global CAR-T cell therapy market.

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Global CAR-T cell therapy Market: Companies Mentioned in Report

Right now, only two companies namely Novartis AG and Gilead Sciences, Inc. have already initiated the process of commercializing their products of CAR-T cell therapy. These two companies jointly hold most of the global CAR-T cell therapy market. However, there are products of other companies that are lying in the pipeline for approval. Other market players comprise Amgen, Inc., Juno Therapeutics, Eli Lilly and Company, Fate Therapeutics, Inc., Sorrento Therapeutics, and Johnson & Johnson.

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CAR-T Cell Therapy Market Segmentation and Forecast Analysis up to 2027 - BioSpace

Ori is focused on bringing automated manufacturing to the cell and gene therapy (CGT) industry, while the CTMCis a joint venture between National Resilience Inc and MD Anderson Cancer Center.

The alliance is aimed at speeding up process development, clinical implementation, and commercialization of novel cell therapies.

It will leverage Oris technology platform with CTMCs manufacturing capabilities and, in the first instance, will focus on a cell therapy invented by Dr Sattva Neelapu, professor of lymphoma/myeloma at MD Anderson.

Ori and CTMC have specifically partnered with the goal of increasing patient access to potentially life-saving cell therapies. This partnership will have a considerable impact on the cell therapy field by implementing innovative, digitally enabled process discovery and automated manufacturing solutions, said Jason C Foster, CEO, Ori Biotech.

Last week we reported on how the CGT manufacturing tech innovator had expanded its global footprint and team, with it executing its strategic plan following the US$100m it raised in its Series B funding round earlier this year.

Ori has invested in high-level expertise, in personnel, having recruited across digital infrastructure, data analytics, software, manufacturing, and quality, and in facilities globally, with the goal of rapid transition into the launch of its CGT manufacturing platform.

The developer has boosted its senior leadership team with several new appointments, including three directors, to support further growth and push the commercialization of its platform, along with further expanding its Science Advisory Board (SAB), appointing CGT experts, Isabelle Rivire and Jason Bock.

It has also opened a new facility in Princeton, New Jersey, US and will be opening a new technology center, in Cambridge, UK.

Commenting on those new recruits and SAB members, Foster told BioPharma-Reporter: As recent data demonstrates, there is an urgent need for increased access to cell and gene therapies, with less than 2% of eligible patients globally gaining access to marketed cell therapies and 20% of those patients who are selected for treatment still die while waiting for their treatment to be manufactured. It is imperative that we address these access issues quickly and there is no time to waste.

"Our new team members add to our strengths across all domains including biology, engineering, data science, manufacturing, supply chain and commercial. Typically, it has taken 10+ years for manufacturing platforms like Oris to get to market, we hope to bring the first generation of our platform to market in early 2024, less than five years post our first round of funding in 2019.

The CEO said the companys Lightspeed Early Access Program (LEAP) is an important step toward commercial launch. It is a very exciting time as we have several partners directly engaging with our beta platform and testing it with their protocols.

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Ori Biotech and CTMC team up on cell therapy delivery - BioPharma-Reporter.com

Adverum Biotechnologies, Inc. (ADVM Quick QuoteADVM - Free Report) announced that the first patient was dosed in the phase II LUNA study evaluating its lead product candidate ixoberogene soroparvovec (Ixo-vec) for the treatment of wet age-related macular degeneration (wet AMD). Shares of this clinical-stage entity were up 2.83% on Sep 14.

Ixoberogene soroparvovec (Ixo-vec) is Adverums clinical-stage gene therapy product candidate, currently being developed for the treatment of wet AMD. Ixo-vec utilizes a proprietary vector capsid, AAV.7m8, carrying an aflibercept coding sequence under the control of a proprietary expression cassette.

While most ophthalmic gene therapies require surgery to administer the gene therapy under the retina (sub-retinal approach), Ixo-vec is designed to be administered as a one-time intravitreal (IVT) injection to deliver long-term efficacy, reduce the burden of frequent anti-vascular endothelial growth factor (VEGF) injections, optimize patient compliance and improve vision outcomes for patients with wet AMD.

LUNA trial is a multicenter, double-masked, randomized, parallel-group study evaluating two doses of Ixo-vec, including the 2x10^11 vg/eye dose and a new, lower 6x10^10 vg/eye dose for wet AMD. The study will enroll 72 participants equally across two doses and four prophylactic steroid regimens in approximately 40 sites in the United States and Europe.

Specific regimens being evaluated include topical Durezol (difluprednate), IVT Ozurdex (dexamethasone) or a combination of either topical Durezol or IVT Ozurdex with oral prednisone to establish a prophylactic regimen with minimal need for inflammation management post prophylaxis.

Per management, LUNA will explore a new, lower 6E10 dose will reinforce the robust, durable efficacy and safety profile already demonstrated with the 2E11 dose in the OPTIC study.

The primary endpoints include the mean change in best corrected visual acuity (BCVA) from baseline to one year, and the incidence and severity of adverse events. Key secondary objectives include the mean change in central subfield thickness (CST) from baseline to one year and assessment of the effectiveness of prophylactic steroid regimens on minimizing inflammation.

Additionally, LUNA will assess aflibercept protein expression starting at week 10 and include an interim analysis at week 26. The study participants will also have the option to enroll in a long-term extension study. Interim data is anticipated in 2023.

The stock has declined 38.1% so far this year compared with the industrys decrease of 23.1%.

Image Source: Zacks Investment Research

The successful development of the candidate will significantly boost this clinical-stage player, considering the potential of Ixo-vec to meaningfully reduce the treatment burden of frequent anti-VEGF injections for wet AMD patients.

The FDA granted Ixo-vec a Fast Track designation to treat wet AMD. In June 2022, the European Medicines Agency (EMA) granted ADVM-022 a Priority Medicines (PRIME) status for wet AMD.

Aflibercept is the active lead component in Regenerons (REGN Quick QuoteREGN - Free Report) Eylea to treat wet AMD. Aflibercept injection 2mg is approved under the brand name Eylea for treating patients with wAMD, macular edema following retinal vein occlusion (RVO), diabetic macular edema (DME) and diabetic retinopathy (DR). Eylea is one of the leading drugs for wAMD.

Regeneron has a collaboration agreement with Bayer AG (BAYRY Quick QuoteBAYRY - Free Report) for Eylea. While Regeneron records net product sales of Eylea in the United States, Bayer records net product sales of the drug outside the United States. REGN records its share of profits/losses in connection with the sales of Eylea outside the United States.

Adverum currently carries a Zacks Rank #3 (Hold). A better-ranked stock in the biotech sector is Dynavax (DVAX Quick QuoteDVAX - Free Report) , which carries a Zacks Rank #2 (Buy) at present. You can see the complete list of todays Zacks #1 Rank (Strong Buy) stocks here.

Dynavaxs earnings estimates have increased to $1.73 from $1.14 for 2022 over the past 60 days. Earnings of DVAX surpassed estimates in two of the trailing four quarters and missed the mark in the remaining two, the average beat being 70.57%.

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Adverum (ADVM) Doses First Patient in the Mid-Stage wAMD Study - Zacks Investment Research

So far, Bcells havent gotten the same attentionindeed, genetically engineered versions have never been tested in a human. Thats partly because engineering B cells is not that easy, says Xin Luo, a professor at Virginia Tech who in 2009 demonstrated how to generate B cells that have an added gene.

That early work, carried out at Caltech, explored whether the cells could be directed to make antibodies against HIV, perhaps becoming a new form of vaccination.

While that idea didnt pan out, now biotech companies like Immusoft, Be Biopharma, and Walking Fish Therapeutics want to harness the cells as molecular factories to treat serious rare diseases. These cells are powerhouses for secreting protein, so thats something they want to take advantage of, says Luo.

Immusoft licensed the Caltech technology and got an early investment from Peter Thiels biotech fund, Breakout Labs. Company founder Matthew Scholz, a software developer, boldly predicted in 2015 that a trial could start immediately. However, the technology the company terms immune-system programming didnt turn out to be as straightforward as coding a computer.

Ainsworth says Immusoft had to first spend several years working out reliable ways to add genes to B cells. Instead of using viruses or gene editing to make genetic changes, the company now employs a transposona molecule that likes to cut and paste DNA segments.

It also took time to convince the FDA to allow the trial. Thats because its known that if added DNA ends up near cancer-promoting genes, it can sometimes turn them on.

The FDA is concerned if you are doing this in a B cell, could you develop a leukemia situation? That is something that they are going to watch pretty closely, says Paul Orchard, the doctor at the University of Minnesota who will be recruiting patients and carrying out the study.

The first human test could resolve some open questions about the technology. One is whether the enhanced cells will take up long-term residence inside peoples bone marrow, where B cells typically live. In theory, the cells could survive decadeseven the entire life of the patient. Another question is whether theyll make enough of the missing enzyme to help stall MPS, which is a progressive disease.

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A new gene therapy based on antibody cells is about to be tested in humans - MIT Technology Review

WAKEFIELD, Mass., September 07, 2022--(BUSINESS WIRE)--Myrtelle Inc., ("Myrtelle" or the "Company"), a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced that the European Medicines Agency (EMA) has classified the Company's lead gene therapy product candidate, rAAV-Olig001-ASPA for the treatment of Canavan disease, as an Advanced Therapy Medicinal Product (ATMP), specifically a Gene Therapy Medicinal Product (GTMP). ATMP classification, which is determined by the Committee for Advanced Therapies (CAT), was established to regulate cell and gene therapy and tissue engineered medicinal products, support development of these products, and provide a benchmark for the level of quality compliance for pharmaceutical practices. As a designated GTMP product, rAAV-Olig001-ASPA will follow the Centralized Procedure through the EMA and benefit from a single evaluation and authorization process. Additional benefits established through the ATMP regulation include pathways for Scientific Advice and significant fee reductions for such advice.

rAAV-Olig001 is a novel vector from a class of recombinant AAVs (rAAVs) that selectively target oligodendrocytes the cells in the brain responsible for producing myelin, the insulating material that enables proper function of neurons and makes up the brains white matter. The Companys lead program is in Phase 1/2 clinical development for Canavan disease (CD) a fatal childhood genetic disorder characterized by the degeneration of the white matter in the brain. The production of myelin is affected in CD due to a mutation in the Aspartoacylase gene (ASPA) leading to deficiency in Aspartoacylase enzyme (ASPA). The oligodendrocyte-targeted gene therapy using the rAAV-Olig001 vector is intended to restore ASPA function, thus enabling metabolism of N-Acetylaspartic Acid (NAA), a neurochemical abundant in the brain, and supporting myelination. Myrtelle entered into an exclusive worldwide licensing agreement with Pfizer Inc. in 2021 to develop and commercialize this novel gene therapy for the treatment of CD.

Story continues

In addition to ATMP classification, rAAV-Olig001-ASPA has been granted US Orphan Drug, Rare Pediatric Disease, and Fast Track designations by the FDA which support the Companys mission to provide treatments for patients with CD. "The designation by the EMA of rAAV-Olig001-ASPA as a Gene Therapy Medicinal Product as a potential treatment for patients with Canavan disease provides important benefits in the development of this innovative therapy. The ATMP classification will facilitate discussions with the EMA as part of our strategy to seek product registration in the EU," said Nancy Barone Kribbs, PhD, Senior Vice President of Global Regulatory Affairs at Myrtelle.

ABOUT MYRTELLE

Myrtelle Inc. is a gene therapy company focused on developing transformative treatments for neurodegenerative diseases. The company has a proprietary platform, intellectual property, and portfolio of programs and technologies supporting innovative gene therapy approaches for neurodegenerative diseases. Myrtelle has an exclusive worldwide licensing agreement with Pfizer for its lead program in Canavan disease. For more information, please visit the Companys website at: http://www.myrtellegtx.com.

ABOUT CANAVAN DISEASE

Canavan disease (CD) is a fatal childhood genetic brain disease in which mutations in the Aspartoacylase gene (ASPA) prevent the normal expression of Aspartoacylase (ASPA), a critical enzyme produced in oligodendrocytes that breaks down the neurochemical N-Acetylaspartate (NAA). When not properly metabolized by oligodendrocytes, NAA accumulates in the brain and negatively affects bioenergetics, myelin production, and brain health. CD patients are impacted at birth but may appear normal until several months old when symptoms begin to develop. Poor head control, abnormally large head size, difficulty in eye tracking, excessive irritability, severely diminished muscle tone, and delays in reaching motor milestones, such as rolling, sitting, and walking, are the typical initial manifestations of CD. As the disease progresses, seizures, spasticity, difficulties in swallowing, and overall muscle deterioration emerge with most affected children developing life-threatening complications by approximately 10 years of age. Currently, there are no cures for CD and only palliative treatments are available. More information on Myrtelles clinical study in Canavan disease can be found on https://clinicaltrials.gov/ under the identifier NCT04833907 or by emailing PatientAdvocacy@MyrtelleGTX.com.

Forward-Looking Statements

This press release contains forward-looking statements. Words such as "may," "believe," "will," "expect," "plan," "anticipate," "estimate," "intend" and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. Forward-looking statements are based upon current estimates and assumptions and include statements regarding rAAV-Olig001-ASPA as a potential treatment for patients with Canavan disease. While Myrtelle believes these forward-looking statements are reasonable, undue reliance should not be placed on any such forward-looking statements, which are based in information available to us on the date of this release. These forward-looking statements are subject to various risks and uncertainties, many of which are difficult to predict, that could cause actual results to differ materially from current expectations and assumptions from those set forth or implied by any forward-looking statements. Important factors that could cause actual results to differ materially from current expectations include, among others, Myrtelles program demonstrating safety and efficacy, as well as results that are consistent with prior results, the ability to generate the data needed for further development of this novel gene therapy in the patients with CD, and the ability to continue its trials and to complete them on time and achieve the desired results. All forward-looking statements are based on Myrtelles expectations and assumptions as of the date of this press release. Actual results may differ materially from these forward-looking statements. Except as required by law, Myrtelle expressly disclaims any responsibility to update any forward-looking statement contained herein, whether as a result of new information, future events or otherwise.

View source version on businesswire.com: https://www.businesswire.com/news/home/20220907005109/en/

Contacts

Media:Jordana HolovachHead of Communications and CommunityMyrtelle Inc.781-621-2797 Ext. 102jholovach@myrtellegtx.com

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Myrtelles rAAV-Olig001-ASPA Gene Therapy Candidate for Canavan Disease Receives Advanced Therapy Medicinal Product Classification from the European...

Repeated muscle injections with Engensis (VM202), Helixmiths investigational non-viral gene therapy, were generally safe and well-tolerated in people with amyotrophic lateral sclerosis (ALS), according to top-line data from a Phase 2a clinical trial.

While the sample size was too small to determine the therapys efficacy, muscle biopsies were collected and will be examined to further evaluate the underlying mechanisms of Engensis.

These data suggest that high dose, repeated treatments of Engensis, were safe and well tolerated, providing a great deal of flexibility in designing dosing schemes for future clinical studies, Helixmith stated in a company press release.

Trial analysis will continue once the full dataset is available, and the company plans to present such findings at a future conference. The next steps for Engensis development will be determined at that time.

Engensis is a non-viral gene therapy that uses Helixmiths proprietary small circular DNA molecule to deliver the hepatocyte growth factor (HGF) gene to cells in the muscle environment.

HGF provides instructions to produce a protein of the same name that helps the body form new blood vessels, prevents muscle loss, and promotes the growth and survival of nerve cells. The therapyis delivered via intramuscular (into-the-muscle) injections.

Helixsmith believes that by increasing HGF production, Engensis has the potential to promote nerve cell and muscle regeneration, thereby countering the progressive loss of motor control that characterizes ALS.

The therapy has been granted orphan drug and fast track designations by the U.S. Food and Drug Administration, both of which are intended to speed its clinical development and regulatory review.

A previous open-label Phase 1/2 trial (NCT02039401) found that four once-weekly intramuscular injections of Engensis (to a total dose of 64 mg) were safe and well-tolerated among 18 ALS patients. Signs that the therapy could slow disease progression were also observed.

These promising findings prompted the launch of a placebo-controlled Phase 2a trial, called REViVALS-1A (NCT04632225), which began patient enrollment last year. A total of 18 ALS patients experiencing motor symptoms in their limbs for four years or less were recruited at four sites in the U.S. and one in Korea.

Participants were randomized in a 2:1 ratio to receive three cycles of either Engensis or a placebo: at studys start, at two months, and at four months. Each cycle consisted of two days of injections to upper and lower limb target muscles, spaced two weeks apart (64 mg total of Engensis or a placebo).

This meant that Engensis-treated patients received a total of 192 mg of medication over the four-month period. All participants were monitored for six months from the studys start.

The trials main goal was to assess the safety and tolerability of Engensis, while efficacy measures were included as exploratory outcomes. These included changes in disability, muscle and lung function, survival, ALS-specific health-related quality of life, and the levels of muscle shrinkage biomarkers.

Top-line data showed that the investigational treatment was generally safe and well-tolerated, with no difference in the frequency of adverse events observed between the Engensis and placebo groups (83% for each).

One case of bronchitis a condition characterized by inflammation in the main airways of the lungs due to infection was observed in the Engensis group but was determined unrelated to treatment.

Injection site reactions were reported by 50% of Engensis-treated patients and 66.7% of those in the placebo group. Most of these reactions were mild or moderate in severity and temporary; no participant discontinued treatment due to the number of injections.

According to Helixmith, efficacy was unable to be evaluated due to the fact that four participants dropped out early from the small study.

Still, muscle tissue biopsies were obtained from injection sites to undergo analyses of muscle atrophy (shrinkage) biomarkers and others.

Since data on Engensis underlying mechanisms have been largely based on animal models, these results are expected to provide valuable information on the understanding of the mechanisms of actions of Engensis, and its effects on the [activity] of human genes, which will greatly help in the development of innovative medicines, the company stated in the release.

Helixmith greatly appreciates the generous and eager participation of the ALS patients, the company added.

Engensis is also being investigated across a range of conditions associated with deficits in circulation, and nerve and/or muscle damage, such as diabetic neuropathy, coronary artery disease, and Charcot-Marie-Tooth disease.

More than 500 patients have been treated with Engensis to date across 10 clinical trials and six different diseases, according to Helixmith. Data from these studies have also supported the therapys favorable safety profile and its ability to increase HGF production.

Link:
Engensis Gene Therapy for ALS Found Safe in Small Phase 2a Trial |... - ALS News Today

Sangamo Therapeutics is continuing to enroll and treat patients with a rare genetic condition called Fabry disease in an early clinical trial of a gene therapy it is developing, announcing Tuesday updated results from the first 11 study participants.

Data to date has shown Sangamos medicine to be safe, with no serious treatment-related adverse reactions, and suggests the gene therapy is working as intended. As a result, Sangamo has begun the dose expansion phase of the trial, enrolling new patients to receive the fourth and highest dose tested in the initial dose escalation cohort.

Sangamo, which has a pipeline of experimental cell, gene replacement and gene editing therapies, is already planning for a potential Phase 3 study, should results from the current trial continue to prove positive. It, along with Freeline Therapeutics, have the most advanced gene therapies in clinical development for Fabry, which is one of an array of inherited conditions known as lysosomal storage disorders.

In Fabry, mutations in a gene called GLA lead to low levels of an enzyme thats needed to prevent the buildup of a certain toxin in cells, causing a constellation of symptoms that over time can become severe and life-threatening. Sangamos therapy is designed to deliver a functional copy of the GLA gene into the body via a type of modified virus that acts as a courier of sorts.

The updated trial results released Thursday show that, among the five earliest treated patients, enzyme levels rose to several times a normal average. In three, enzyme levels were 10 to 17 times higher. Notably, in one patient with the highest toxin level pre-treatment, the gene therapy led to a 40% decline in toxin levels within 10 weeks after dosing.

Five patients have been able to discontinue standard drugs, which in the case of Fabry is an enzyme replacement therapy that must be taken chronically to control toxin levels.

According to Sangamo, there were no treatment-related side effects rated by investigators as more significant than mild, and no patient experienced elevations in liver enzymes that can sometimes signal broader safety concerns.

In the next part of the trial, Sangamo plans to enroll up to six participants in six different cohorts.

Sangamo and Freeline were previously joined by Avrobio in developing a Fabry gene therapy. But in February Avrobio announced it would stop work on its program after disappointing findings and prioritize research elsewhere. Amicus Therapeutics and UniQure also have Fabry gene therapy programs, although Amicus recently had to pivot after plans to spin out its gene therapy business fell apart.

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Sangamo presses ahead with Fabry disease gene therapy - BioPharma Dive

Tegan Taylor: There are a few things in life that are just inevitable; death, taxes, the genes you're born with. At least, that has been the case for pretty much every generation up until now. Gene therapies have the potential to change the trajectory of disease, and I've been talking to two people on the frontline of that shift.

Until about three years ago, Robert Lamberth had a disease that was incurable. I mean, it was literally in his genes.

Robert Lamberth: Not as a newborn, but yes, very, very young when I had my first bleed. Three, I think it might have been for me, back in the early '80s it was, a long time ago now.

Tegan Taylor: When he was born, he inherited a certain recessive gene that stopped his body from producing one of the essential factors you need for your blood to clot.

Robert Lamberth: Bleeding internally into my major weight-bearing joints, so ankles and knees. And as I got older, I'd have more odd bleeding into muscles in my legs and parts of my stomach and those sorts of things, so it was a little bit more serious when you have large muscle bleeds. The pressure of the bleeding can affect your organs, so that's quite serious.

Tegan Taylor: Managing haemophilia A is miles easier than it was a couple of decades ago. When he was little, Robert needed intravenous injections of his missing clotting factor, given in a hospital. When he got older, he didn't need to go to hospital anymore. Regular injections of the clotting factor were a feature of his life all the way through into his 30s. But not anymore.

John Rasko: We dream of cures in gene therapy but hesitate to use the word

Tegan Taylor: For decades, John Rasko has been chasing ways to change people's fates.

John Rasko: For the last 20-plus years we've been doing clinical trials using viral vectors to transfer a gene into humans for a therapeutic purpose.

Tegan Taylor: Professor Rasko is a haematologist and pathologist who spent much of his career studying genes, stem cells and basically how to hack processes inside the human body. And he is one of many scientists around the world trying to figure out ways of swapping out disease-causing genes in a way that, in time, could be used for pretty much any genetic disease.

John Rasko: When we reflect on rare diseases, it's often worth remarking and reminding ourselves that rare diseases of course by definition are rare, usually less than one in 5,000 or 10,000 people, but collectively rare diseases are very common when you add them all up because there are many thousands of them, lead to a burden of disease such as the commonality of diabetes or even some forms of cancer. So the problem is that of all the rare diseases, which some people say are more than 4,000 affecting humans, 80% of those rare diseases have a genetic basis. And of those diseases, only 5% have a specific therapy. So this is an incredible unmet need in human health.

Tegan Taylor: And the solution he and his colleagues have come up with might sound a bit familiar. It works in a similar way to the Covid vaccine made by AstraZeneca. It uses a harmless virus to take a genetic message into the body.

John Rasko: And that vector system is used to then ferry that genetic payload intravenously to the liver where it takes up residence, and hopefully after a single injection, corrects that person's genetic abnormality for the rest of their life. It's unimaginable, but a single injection can alter the course of a genetic disease that would otherwise affect a person from birth to death.

Tegan Taylor: Robert was part of the clinical trial Professor Rasko was involved in, testing the gene therapy.

Robert Lamberth: It would be three years ago now in May 2019 when I had that one single dose of the good stuff, and then that clearly worked its magic and now I'm growing my own factor VIII. I've had one breakthrough bleed.

Gene therapy for me, Tegan, has been quite revolutionary, so from a position of having 0.5% of clotting factor in my blood, I'm now growing my own factor VIII in my liver and I'm at about 15% clotting factor, which is an extraordinary growth.

Tegan Taylor: In August, Europe granted conditional approval for a haemophilia A therapy like the one Robert received. It hasn't been approved in Australia yet, although we do use gene therapy for other conditions, like spinal muscular atrophy, and genetic causes of blindness.

John Rasko: We are only at the very start of this genetic revolution. There are thousands of genetic diseases that affect humans, and we've only just started scratching the surface of where we can go with these gene-based therapeutics.

Tegan Taylor: Because Robert got the gene therapy as an adult, he's still living with the damage haemophilia A had already done to his body, but that doesn't mean it hasn't been transformative.

Robert Lamberth: I can just do so much more. I can be out there doing everything that I love at work and at play and going to the gym, without fear of having a micro-bleed the next day and being cross and crotchety and painful and grumpy at work, and then it turning into a more major bleed and then having to go and seek therapy, which means even more down-time. The sooner that we could roll out some gene therapy for younger people would be great.

Tegan Taylor: Robert Lamberth, who received gene therapy for haemophilia A, finishing us off there. And we also heard from Professor John Rasko from Royal Prince Alfred Hospital and the Centenary Institute at the University of Sydney.

Norman Swan: It's interesting how things have advanced there, Tegan. A few years ago, not so long ago, gene therapy could have been quite toxic because of the virus that they were using to carry the gene in, and you've got to hit the target, it can't be wasteful, and sometimes the virus did harm in its own right. So it's taken a long time to get that right, but the potential, as John Rasko says, is huge and it goes from cancer through to these inborn errors that you get such as haemophilia A.

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The gene therapy that could transform the lives of millions - ABC News