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 ...

RT Patri Friedman 🌆 (@patrissimo)TLDR: The @MinicircleDNA follistatin gene therapy increased my VO2 max by as much in a week as 3 months of training.I'm sharing this due to the doubt I've seen both from anti-tech media & my skeptical, well-intentioned friends like @yashgaroth & @s_r_constantin.I'm not qualified to evaluate the science & I don't have N=many data, but I can speak as an N=1 who saw significant performance impact from this treatment @farbood, @bryan_johnson, @michaelmalice & others got at @Pros ...

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 ...

Summary of Ocular Therapeutix (OCUL) Conference Call Company Overview - **Company**: Ocular Therapeutix (OCUL) - **Event**: 2025 Conference on May 27, 2025 - **Key Speaker**: Praveen Dugal, Executive Chairman, President, and CEO Core Industry Insights - **Industry**: Ophthalmology, specifically focusing on treatments for retinal diseases - **Market Context**: The anti-VEGF (vascular endothelial growth factor) market has been established for over 20 years, with ongoing unmet needs for longer-duration treatments [7][10] Key Points and Arguments 1. **Need for Innovation**: There is a significant need for improved treatment options in the anti-VEGF market, as nearly 40% of patients drop out of treatment within the first year due to the unsustainability of frequent injections [10][11] 2. **Product Technology**: Ocular Therapeutix's product, ex-Paxley, is a tunable, completely dissolvable hydrogel that has shown to be safe and efficacious based on consistent data [4][5] 3. **Regulatory Pathway**: The company has a clear regulatory path forward, having received Special Protocol Assessment (SPA) from the FDA for their studies, which aligns with the latest FDA guidelines [5][40] 4. **Treatment Paradigm Shift**: ex-Paxley is positioned to potentially replace existing anti-VEGF treatments as a first-line agent due to its efficacy and the ability to provide longer-lasting results [22][23] 5. **Patient Selection**: The company emphasizes the importance of proper patient selection in clinical trials, aiming to identify patients who will benefit most from the treatment [30][31] 6. **Study Design**: The company has designed two complementary studies (SOLAR and SOLA-one) to gather comprehensive data, with a focus on patient stability and response to treatment [44][45] 7. **Market Opportunity**: The market for retinal disease treatments is vast, and Ocular Therapeutix is confident in its ability to capture significant market share due to its innovative product and strong regulatory support [60] Additional Important Insights - **Retention Rates**: The company has reported unprecedented patient retention rates in their studies, which they attribute to the quality of patient selection and the nature of the treatment [54] - **Regulatory Collaboration**: Ocular Therapeutix has established a strong collaborative relationship with the FDA, which has facilitated a smoother regulatory process for their studies [50] - **Future Outlook**: The company is optimistic about the future of ex-Paxley and its potential to dominate the market once the macroeconomic environment stabilizes [59][60] This summary encapsulates the critical insights and developments discussed during the conference call, highlighting Ocular Therapeutix's strategic positioning within the ophthalmology industry and its innovative approach to addressing unmet patient needs.

Core Insights - Adverum Biotechnologies has initiated the ARTEMIS Phase 3 study to evaluate the efficacy and safety of Ixo-vec for treating wet age-related macular degeneration (AMD) [1][4] - The trial aims to demonstrate that a single injection of Ixo-vec is non-inferior to the standard treatment with aflibercept, potentially transforming the treatment landscape for wet AMD patients [5][10] Company Overview - Adverum Biotechnologies is a clinical-stage company focused on gene therapy to treat prevalent ocular diseases, aiming to establish a new standard of care [11][12] - The company is developing Ixo-vec, a one-time gene therapy designed to deliver long-term efficacy and reduce the burden of frequent anti-VEGF injections for wet AMD patients [10][11] Study Design - The ARTEMIS trial is a randomized, double-masked, sham-controlled study involving approximately 284 patients, including both treatment-naïve and previously treated individuals [7][8] - The primary endpoint is the mean change in best corrected visual acuity (BCVA) from baseline at one year, with a non-inferiority margin of -4.5 letters [2][8] Treatment Context - Wet AMD affects around 20 million individuals globally and is a leading cause of blindness in those over 65 years of age [9] - Current treatment requires frequent injections, with up to 42% of patients discontinuing treatment after two years, leading to poor long-term vision outcomes [4][9] Ixo-vec Characteristics - Ixo-vec utilizes a proprietary vector for a one-time intravitreal injection, aiming to provide stable therapeutic levels of anti-VEGF and improve patient compliance [10][11] - The therapy has received Fast Track and Regenerative Medicine Advanced Therapy designations from the FDA, indicating its potential as a transformative treatment option [10]

Core Insights - Adverum Biotechnologies has initiated the ARTEMIS Phase 3 study to evaluate the efficacy and safety of Ixo-vec for treating wet age-related macular degeneration (AMD) [1][4] - The trial aims to demonstrate that a single injection of Ixo-vec is non-inferior to the standard treatment with aflibercept [2][5] Study Design - ARTEMIS is a US-based, randomized, double-masked, sham-controlled trial involving approximately 284 patients, including both treatment-naïve and previously treated individuals [2][7] - The primary endpoint is the mean change in best corrected visual acuity (BCVA) from baseline at one year, with a non-inferiority margin of -4.5 letters [2][8] - All participants will receive three loading doses of aflibercept before administration of Ixo-vec [2][8] Significance of Ixo-vec - Ixo-vec is positioned as a potential best-in-class gene therapy that could provide long-term efficacy and reduce the treatment burden associated with frequent anti-VEGF injections [5][10] - The therapy is designed for a one-time intravitreal injection, which is expected to optimize patient compliance and improve vision outcomes [10][11] Unmet Medical Need - There is a significant unmet need for longer-acting therapies for wet AMD, with data indicating that up to 42% of patients discontinue treatment after two years, leading to poor long-term vision outcomes [4][9] - The current standard of care requires lifelong repeated injections, highlighting the potential impact of Ixo-vec in reducing treatment frequency [9][10] Regulatory Designations - Ixo-vec has received Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA, as well as PRIME designation from the EMA [10][11]