They didn’t just innovate — they rewrote the future of medicine.Jennifer Doudna — CRISPR pioneer + Nobel Prize winnerJudy Faulkner — built modern electronic health recordsMartine Rothblatt — from SiriusXM to biotech saving livesSuma Krishnan — first topical gene therapy creatorSee the full #Forbes250 America's Greatest Innovators list: https://t.co/S1qgkoTqmt (Jennifer Doudna By Cody Pickens For Forbes; Judy Faulkner By Guerin Blask For Forbes; Martine Rothblatt By Martin Schoeller; Suma Krishnan By Jamel T ...

Core Insights - Voyager Therapeutics is focusing on 2025 as the "year of tau," with two key assets targeting tau-related conditions [2] - The company anticipates tau PET imaging data from a multiple ascending dose study by the end of the year [2] - A gene therapy asset aimed at tau knockdown, similar to BIIB080, is expected to have significant readouts in the mid-year timeframe [2] Group 1 - The first asset is a C-terminal antibody targeting tau, with imaging data expected soon [2] - The second asset is a gene therapy for tau knockdown, which is part of the company's broader strategy [2] - Voyager is introducing newly discovered blood-brain barrier (BBB) penetrant capsids into clinical trials for the first time this year [3] Group 2 - The company has two assets utilizing these capsids: the tau knockdown gene therapy and a partnered program for Friedreich's ataxia [3] - Voyager aims to establish proof of concept for the efficacy of its capsids in producing gene expression [3]

Lucas. His soul chose this body, chose us as parents. For the world to care about rare diseases, people's hearts need to be changed.You know, we can't just have money be thrown into the problem. 831 is blindfolded and can no longer see what's going on. Beast games application.Mr. . beast, I don't have a job right now. Like I think I can apply $10 million.It's all a miracle. But it's also tragic to think that I have to go in the largest game show ever for people to know about my son. Keep going.Good job. Gre ...

They didn’t just innovate — they rewrote the future of medicine.Jennifer Doudna — CRISPR pioneer + Nobel Prize winnerJudy Faulkner — built modern electronic health recordsMartine Rothblatt — from SiriusXM to biotech saving livesSuma Krishnan — first topical gene therapy creatorSee the full #Forbes250 America's Greatest Innovators list https://t.co/S1qgkoTqmt (Jennifer Doudna By Cody Pickens For Forbes; Judy Faulkner By Guerin Blask For Forbes; Martine Rothblatt By Martin Schoeller; Suma Krishnan By Jamel To ...

Core Viewpoint - The FDA supports mRNA vaccines but believes that U.S. taxpayers should not bear the financial burden of their development, as companies involved can fund their own research [2][8]. Group 1: FDA's Position on mRNA Vaccines - FDA Commissioner Marty Makary defended the agency's stance on mRNA vaccines, stating that the FDA has approved two mRNA vaccines for RSV [3]. - The FDA is reviewing a Moderna flu vaccine, indicating ongoing support for mRNA technology [1][2]. Group 2: Funding Decisions and Rationale - The U.S. Department of Health and Human Services (HHS) cut funding for mRNA vaccine research, with Makary explaining that companies like Moderna and Pfizer, which made over $50 billion, can afford their own research [5][8]. - The HHS's decision to defund mRNA vaccine development was influenced by a belief that the vaccines were not effectively protecting against respiratory infections, despite contrary scientific evidence [5][6]. Group 3: Future Research Directions - Makary emphasized the need for government funding to focus on underfunded areas such as rare diseases and bespoke conditions, rather than mRNA vaccine research [9].

Company Overview - Solid Biosciences is a precision genetic medicine company with approximately 120 employees, primarily focusing on gene therapies [2] - The company has three main drug candidates: one for Duchenne Muscular Dystrophy, another for Friedreich’s Ataxia, and a third for Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT) [2] Product Pipeline - The next program in development targets dilated cardiomyopathies, specifically TNNT2 [3] - Solid Biosciences is building a comprehensive capsid library and platform to enhance gene therapy delivery, focusing on capsids, promoters, dual plasmids, and manufacturing purity [3]

Industry Overview - The biotech industry had a strong performance in 2025, outperforming NVIDIA, with continued enthusiasm into 2026 [5] - There are concerns regarding the FDA's regulatory environment, described as chaotic, which may impact drug approvals [5][9] FDA Regulatory Environment - Richard Pasdur, a former senior FDA executive, expressed concerns about the breach of the firewall between political appointees and drug reviewers, indicating potential risks for the pharma industry [7] - The FDA's current commissioner, Marty Makary, aims to speed up drug approvals, but there are conflicting signals regarding the actual operating reality [9] Company Earnings Predictions Eli Lilly (Ticker: LLY) - Expected earnings per share range from $6.99 to $7.86, with a consensus estimate of $7.48 and revenue of $17.85 billion, indicating over 30% year-over-year earnings growth [15] - Analysts predict a likely earnings beat due to a history of exceeding expectations [15][16] Novo Nordisk (Ticker: NVO) - Earnings per share expectations are between $0.89 and $0.90, with revenue projected at $11.96 billion, showing a flat to slightly down trend [17] - Analysts express a higher possibility of a miss compared to Eli Lilly, citing recent struggles and a new CEO's uncertain direction [17][19] Twist Bioscience (Ticker: TWST) - Reported revenue of $104 million for Q1 2026, consistent with preliminary results, though still unprofitable [20] - The company has shown consistent revenue growth despite industry challenges, reducing cash burn and moving towards profitability [20]

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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].