Genespire Presents Promising Dosing Data for First-in-Human Gene Therapy in Methylmalonic Acidemia
• Genespire's preclinical research demonstrates that immune-shielded lentiviral vectors (ISLVs) effectively treat methylmalonic acidemia in mouse models, with codon-optimized versions showing higher efficacy at lower doses.
• The study revealed dose-dependent improvements across three dosing levels, with evidence that even the lowest dose provided benefits due to selective advantage of treated cells in the liver.
• Results from humanized mouse models indicate that CD47-enriched vectors achieve comparable efficacy at substantially lower doses, providing critical insights for upcoming Phase I clinical trials of GENE202 planned for 2026.
MeiraGTx Unveils Breakthrough Gene Therapies for Obesity and Neurodegenerative Diseases at ASGCT 2025
• MeiraGTx presented four posters at ASGCT 2025, showcasing advancements in gene therapies for severe pediatric obesity, ALS, and frontotemporal dementia, highlighting their innovative approach to addressing significant unmet medical needs.
• The company's optimized AAV-BDNF vector for obesity demonstrated remarkable efficacy in preclinical models, achieving up to 40% weight loss compared to just 12% with semaglutide, while maintaining normal behavioral patterns in treated animals.
• MeiraGTx has developed novel CAG promoter variants that are both shorter and up to 15-fold more potent than traditional promoters, potentially enabling lower therapeutic doses and improved safety profiles for future gene therapy applications.
Leap Therapeutics Halves Workforce and Refocuses Cancer Drug Development Amid Market Challenges
• Leap Therapeutics has announced a significant restructuring, reducing its workforce by approximately 50% and narrowing the development focus of its lead cancer drug candidate in response to challenging market conditions.
• The strategic pivot aims to extend the company's cash runway while concentrating resources on the most promising clinical applications of its lead oncology asset, potentially improving its chances for regulatory success.
• This move follows similar restructuring trends across the biotech sector, with companies like Arcturus, NGM Bio, and Erasca all recently announcing staff reductions and pipeline reprioritizations to navigate the difficult funding environment.
Next-Generation Gene Therapies: Evolving Beyond Viral Vectors Towards More Affordable, Sustainable Solutions
• Despite 32 approved gene therapies globally, the industry faces significant challenges in safety, efficacy, and affordability, prompting development of novel delivery systems beyond traditional viral vectors.
• Companies are advancing non-viral delivery platforms including exosomes, lipid nanoparticles, and hydrophilic nanoparticles that offer cost-effective alternatives with reduced immunogenicity and potential for repeat dosing.
• Next-generation gene editing technologies like Prime Editing and CRISPR variants are emerging as more precise alternatives to traditional CRISPR-Cas9, with Prime Medicine's PM359 for chronic granulomatous disease advancing to clinical trials.
MeiraGTx and Hologen AI Form $430 Million Strategic Collaboration to Advance Parkinson's Disease Gene Therapy
• MeiraGTx has secured a strategic collaboration with Hologen AI that includes a $200 million upfront payment and the formation of a joint venture with an additional $230 million in committed capital to advance AAV-GAD gene therapy for Parkinson's disease.
• The partnership creates the first neuro-AI clinical drug development company, combining MeiraGTx's gene therapy expertise with Hologen's AI technology to transform CNS drug development for neurodegenerative and neuropsychiatric disorders.
• Recent clinical data showed AAV-GAD therapy demonstrated significant benefits in Parkinson's disease patients, with meaningful improvements in motor function (UPDRS Part 3) and quality of life measures (PDQ-39).
Biotech Deal Landscape: February-March 2025 Sees Surge in Partnerships Across Multiple Therapeutic Areas
• The first quarter of 2025 witnessed significant biotech partnership activity, with Eli Lilly, AstraZeneca, and Novo Nordisk emerging as top collaborators in deals worth billions across small molecules, antibodies, and RNA therapeutics.
• February 2025 featured notable acquisitions including Novartis's $2.15 billion buyout of Anthos Therapeutics, while March saw AstraZeneca acquire Belgian biotech EsoBiotec and Bristol Myers Squibb purchase 2seventy bio for $286 million.
• Obesity therapeutics gained significant traction in March 2025, with AbbVie entering the field through a $350 million upfront deal with Gubra for an amylin analog, while Roche partnered with Zealand Pharma on petrelintide in a deal worth up to $5.25 billion.
Mass General Brigham Reports Universal Patient Response in Corneal Stem Cell Therapy Trial
• Mass General Brigham's corneal stem cell therapy trial demonstrates positive outcomes across all enrolled patients, marking a significant advancement in regenerative eye treatment.
• The breakthrough coincides with promising developments in gene-based eye care, including MeiraGTx's gene therapy showing vision improvements in congenitally blind children.
• Emmecell's non-surgical cell therapy candidate EO2002 achieves positive topline results in Phase I extension trial for corneal edema treatment.
MeiraGTX's Gene Therapy Shows Promising Visual Benefits in Children with Rare Genetic Blindness
• Landmark clinical trial demonstrates significant visual improvements in all four young children treated with MeiraGTX's rAAV8.hRKp.AIPL1 gene therapy for Leber congenital amaurosis 4 retinal dystrophy.
• Additional seven children in the expanded trial cohort also showed benefits from the single-dose treatment, with visual acuity gains observed within four weeks of administration.
• MeiraGTX plans to submit a Marketing Authorization Application in the UK under exceptional circumstances based on positive results from all 11 treated children.
Breakthrough Gene Therapy Restores Vision in Children with Rare Genetic Blindness
• A groundbreaking gene therapy developed by Moorfields Eye Hospital and MeiraGTx has successfully restored vision in children born with LCA-AIPL1, a severe inherited eye disorder affecting 2-3 in 10 million newborns.
• Clinical trials demonstrated significant vision improvement in all eleven treated children, with some achieving 20/80 vision, enabling them to identify pictures, safely navigate, and engage in visual activities for the first time.
• The treatment must be administered before age four due to irreversible retinal degeneration, with regulatory approval expected within 1-2 years following successful safety and efficacy demonstrations.
AAV Gene Therapy Market Projected to Reach $107.2 Billion by 2035, Growing at 40% CAGR
• The adeno-associated virus (AAV) gene therapy market is expected to surge from $2.7 billion in 2024 to $107.2 billion by 2035, driven by advances in genetics, biotechnology, and personalized medicine.
• Marketed AAV therapies include Luxturna for hereditary retinal dystrophy, Zolgensma for spinal muscular atrophy, and Glybera for lipoprotein lipase deficiency, with numerous promising candidates in late-stage development.
• Improved diagnostic technologies, regulatory support, and significant investment from companies like Spark Therapeutics, Novartis, and BioMarin are accelerating the development of next-generation AAV therapies for previously untreatable genetic disorders.
MeiraGTx's AAV2-hAQP1 Receives FDA RMAT Designation for Radiation-Induced Xerostomia
• MeiraGTx's AAV2-hAQP1 gene therapy has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA for grade 2/3 radiation-induced xerostomia (RIX).
• The RMAT designation includes benefits like Fast Track and Breakthrough Therapy designations, facilitating closer collaboration with the FDA and potentially accelerating approval.
• Phase 1 trial data showed significant improvements in patient-reported outcomes and saliva production with AAV2-hAQP1, supporting the RMAT designation for this debilitating condition.
• The ongoing Phase 2 AQUAx2 trial is designed as a pivotal study, potentially leading to a Biologics License Application (BLA) filing based on MeiraGTx's proprietary manufacturing process.
Gene Therapy Advances: From FDA Clearances to Clinical Trial Progress in Early 2025
• The FDA cleared United Therapeutics' UKidney for US trials, a gene-edited porcine kidney product intended for end-stage renal disease via xenotransplant.
• uniQure received the green light to proceed with the second dose cohort in their Phase 1/2 trial of AMT-162 for SOD1-ALS gene therapy.
• Sarepta Therapeutics' Elevidys showed sustained benefit in ambulatory patients with Duchenne muscular dystrophy in Phase 3 trial results.
Gene Therapy Clinical Trial Pipeline Surges with Over 180 Companies Leading Innovation
• The gene therapy market is experiencing substantial growth, fueled by technological advancements, increased investments, and a deeper understanding of genetic diseases.
• Key players are actively engaging in collaborations, mergers, and acquisitions to enhance their research and development capabilities and broaden their product portfolios.
• Regulatory bodies like the FDA and EMA are expediting the approval process for gene therapies, acknowledging their potential in addressing unmet medical needs.
• Ethical considerations and public perception surrounding gene editing, particularly germline modifications, remain critical factors influencing market dynamics and regulatory policies.
MeiraGTx's AAV-GAD Gene Therapy Shows Promise in Parkinson's Disease Phase I/II Trial
• MeiraGTx's AAV-GAD gene therapy met its primary endpoint, demonstrating safety and tolerability in a Phase I/II trial for Parkinson's disease.
• The high-dose group showed a statistically significant 18-point improvement in the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III score.
• Significant improvements in quality of life were observed in both low- and high-dose groups, as measured by the Parkinson’s Disease Questionnaire (PDQ-39).
• MeiraGTx plans to initiate a Phase III study in 2025, following positive discussions with regulatory bodies in the US, Europe, and Japan.
Pfizer Invests in Molecular Glue Research; Jazz's Zepzelca Shows Promise in Lung Cancer
• Pfizer has invested $49 million in Triana Biomedicines to discover novel molecular glue degraders for oncology, with Pfizer holding an exclusive option to license promising candidates.
• Jazz Pharmaceuticals' Zepzelca, combined with Roche's Tecentriq, demonstrated improved overall survival and progression-free survival in small cell lung cancer patients in the IMforte Phase 3 study.
• MeiraGTx's experimental gene therapy for Parkinson's disease met its primary endpoint in a small clinical trial, showing safety and tolerability, with encouraging signs of efficacy.
• Akeso secured $250 million in private funding to advance global trials of its antibody drugs for cancer and autoimmune diseases, following positive data comparing its drug to Merck's Keytruda.
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