Sarepta said a study designed to confirm the benefits of two of its older drugs for a devastating muscle disease failed, dealing a setback to the company working to recover from a controversy over its gene therapy https://t.co/zxbhNkKhgO ...

Core Viewpoint - Benitec Biopharma Inc. is set to provide an update on the six treated patients from Cohort 1 of its Phase 1b/2a Clinical Study of BB-301, which is being investigated for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD) [1]. Company Overview - Benitec Biopharma Inc. is a clinical-stage biotechnology company based in Hayward, California, focused on developing novel genetic medicines [3]. - The company utilizes a proprietary "Silence and Replace" DNA-directed RNA interference (ddRNAi) platform that combines RNA interference with gene therapy [3]. - This platform aims to create therapeutics that can silence disease-causing genes while delivering wildtype replacement genes in a single administration [3]. - Benitec is developing therapeutics for chronic and life-threatening conditions, including Oculopharyngeal Muscular Dystrophy (OPMD) [3]. Webcast Information - A webcast to discuss the update on the clinical study will take place on November 3, 2025, at 8:00 am EST [2].

Program Overview - YZi Labs launches the second season of EASY Residency incubation program [1] - The program is an eight-week initiative [1] - The program focuses on global collaboration among entrepreneurs in Web3, AI, and Biotech [1] Focus Areas - The program covers areas such as stablecoins and payments [1] - The program covers Web3 creator tools [1] - The program covers agentic AI [1] - The program covers gene therapy [1]

Michelle Ross is the CIO of Stempoint Capital where she runs a biotech fund and she joins us once again now. It's good to see you. Very good to see you too.A reason to have a smile. Yeah. In in this space is what do you think of what Krinsky says.Is it meaningful. It's uh it's very unique set of circumstances that that have led us to this point. And I think what we saw even last week was pretty dynamic in the sense of how much some of these names traded up on the back of an announcement by the administratio ...

Gene Therapy Performance - MinicircleDNA follistatin gene therapy shows potential to increase VO2 max, with one individual reporting an increase comparable to 3 months of training within a week [1] - The individual experienced an almost 50% increase in running endurance with the same effort after the therapy [3] - The individual participated in a reversibility study to turn off the gene therapy and subsequently received the therapy again [3] VO2 Max Data - Baseline VO2max increased by 4.8 ml/kg/min over 2 months of training [4] - VO2max increased by 4.3 ml/kg/min in one week after the gene therapy [4] - VO2max increased by an additional 3.6 ml/kg/min over 3 months of training [4] - The reported VO2max differences exceed the expected 2%-5% per-trial variation of 1-3 ml/kg/min [4] Skepticism and Call to Action - The individual acknowledges skepticism due to the novelty of the treatment and limited public data [5] - The individual encourages others undergoing the therapy to collect and share data [5]

The first ever successful treatment for Huntington's disease, possibly discovered in a UK clinical trial. This is a gene therapy. It's delivered via brain surgery.It slowed the progression of the disease by about 75% in some instances. Huntington's is a brain disease that gets worse over time. It leads to slower thought, slower movement.The lead researcher on this study called it worldch changing. ...

Healthcare & Insurance Industry Focus - A rare genetic disorder, Duchenne Muscular Dystrophy (DMD), affects two brothers, leading to muscle degeneration and reduced life expectancy [3] - The treatment, Elevidys, a one-time dose gene therapy, costs $32 million [4] - Blue Cross Blue Shield's federal employee program initially denied coverage for one of the brothers, Hunter [5][6] - Public and peer pressure, including a letter-writing campaign, influenced the insurance company to revisit the case [7] - Blue Cross Blue Shield approved Hunter's treatment after an appeal [8] Ethical & Social Implications - The case highlights the challenges families face in accessing expensive, potentially life-improving treatments [4][9] - Community support and media attention can play a significant role in influencing healthcare decisions [7][9] - The story underscores the importance of equal access to medical care, especially for siblings with the same condition [1][5] Patient Outcomes & Treatment Efficacy - Gene therapy has shown the potential to slow the progression of DMD [4] - One brother, Noah, experienced positive outcomes after receiving gene therapy, including improved physical abilities [2] - The treatment aims to improve the quality of life and extend the lifespan of individuals with DMD [9]

Company Overview - enGene specializes in nonviral gene therapy, which addresses some of the common shortcomings associated with traditional gene therapies [2] - The company utilizes a unique Special DDX platform to deliver gene therapy through mucosal routes, enhancing its effectiveness [2] Product Features - enGene's technology allows for the delivery of large genetic cargo without limitations, making it versatile for various applications [3] - The products are redoseable, enabling repeated use as necessary, which is a significant advantage in treatment [3] - The nonviral nature of the therapy simplifies manufacturing processes, leading to competitive cost of goods and broader applicability across diseases [3]

Sarepta announced that the company plans to temporarily pause all shipments of its gene therapy to treat Duchenne muscular dystrophy, Elevidys, in a reversal of its prior stance https://t.co/RqYHvvW5xV ...

>> All right. Welcome back, everybody. The Trump administration has cut health research funding to $2.8% billion in May.That's down 28% from April. That's according to data from the Treasury Department. Joining us right now to discuss the implications is former FDA Commissioner Scott Gottlieb.He's 2828. It's 2.8%, not 28. He's a board member with Pfizer and Illumina.Also a CNBC contributor. Doctor Gottlieb, thanks for being here today. First of all, describe what happened.Why was the funding cut and where w ...