Sarepta Therapeutics’ Post

Today Ocugen announced that the Data and Safety Monitoring Board approved enrollment in Cohort 3 as well as Phase 2 initiation in the Phase 1/2 ArMaDa clinical trial for OCU410—a modifier gene therapy candidate being developed for geographic atrophy. We are very enthusiastic about the potential of OCU410 as a potential one-time treatment for GA with a single sub-retinal injection. The currently approved treatments for GA target only the complement pathway and require approximately 6-12 intravitreal injections annually. OCU410 addresses multiple pathways causing dAMD, including complement, lipid metabolism, inflammation, and oxidative stress, potentially providing long-term benefit to patients. Read the full press release here: https://bit.ly/4aLjz6H

 Are you testing therapeutics for ocular toxicity? See how retinal pigment epithelium models can be used to investigate efficacy of gene therapy vectors on both healthy and patient-derived tissues [ link to https://lnkd.in/eQxtFhrf] Speak to our experts to accelerate your research.

Qiming Venture Partners' portfolio company Belief BioMed has presented the results of its Phase 3 clinical trial for BBM-H901, a gene therapy for adults with hemophilia B, at the 2024 American Society of Hematology Annual Meeting. The study highlights BBM-H901's ability to significantly reduce annualized bleeding rates (ABR), rapidly increase FIX activity levels, and maintain stable expression, compared to prophylactic FIX replacement therapy—with excellent safety and tolerability. #GeneTherapy #HemophiliaB #ClinicalTrials #ASH2024 #InnovativeMedicine #BeliefBioMed #QimingPortfolio #QimingHealthcare

In 2024, the biotherapeutics industry made significant progress in transforming disease treatment, one example being the landmark approval of CASGEVY®, the first cell-based gene therapy for the treatment of transfusion-dependent beta-thalassemia. In this Q&A, Dr. Francisca Neethling, Head of Biotherapeutic Services at Eurofins Discovery, shares the highs and lows of the past year, the team’s successes and what to expect in 2025. Read it now: https://lnkd.in/gBfHdEv7

Majda Hadziahmetovic, MD, served as the Lead Principal Investigator on a Phase 1 clinical trial that is showing promising results for patients with geographic atrophy. The trial demonstrated reduced lesion growth, preservation of retinal tissue, and improved visual function. This novel gene therapy could be a game-changer for patients with dry age-related macular degeneration. Read more: https://lnkd.in/egrPeN3W

Join Dr. Craig Kessler and Dr. Guy Young for a 1-hour accredited learning activity on the evolving therapeutic landscape of hemophilia. This activity covers therapies tailored to meet the individual needs of patients with Hemophilia A and Hemophilia B. The faculty share their insights and experiences with clotting factor concentrates, recombinant factors, factor VIII mimetics, rebalancing agents, and gene therapies. They will also discuss the safety and efficacy of late-phase investigational products that have the potential to address unmet needs and further transform patient care. https://lnkd.in/g4G7kvFR

Things are now really starting to accelerate in this space - bring it on ..

NEWS ALERT: Encouraging early data from Tenaya’s MyPEAK™-1 Phase 1b clinical trial for the treatment of MYBPC3-associated hypertrophic cardiomyopathy (#HCM). MyPEAK-1 is an on-going clinical trial evaluating the first gene therapy for the leading genetic cause of HCM to be assessed in humans. Learn more: bit.ly/4gHtv4x #GeneTherapy

For biotech companies embarking on clinical trial planning and execution, finding a CRO to help anticipate and navigate the operational complexities of CAGT therapies is critical. IQVIA Biotech’s multidisciplinary Cell and Gene Therapy Study Management team provides end-to-end solutions to streamline your processes and advance your CAGT clinical trial. Discover more. https://bit.ly/3BVi9Ll #CAGT #clinicaltrials

Did you know that the ATTC network is made up of 4 Advanced Therapy Treatment Centres spread across the UK? ➡️ https://ow.ly/liux50U63c6 Coordinated by the Cell and Gene Therapy Catapult, the ATTC network operates within the NHS framework to address the unique and complex challenges of bringing pioneering advanced therapy medicinal products (ATMPs) to patients. Learn more 👆 #ATTCnetwork #PatientAccess #AdvancedTherapies