Skip to main content Exclusive news, data and analytics for financial market professionalsLearn more aboutRefinitiv US FDA Commissioner Makary says FDA supports mRNA vaccines but US taxpayers should not bear the cost February 23, 20268:15 PM UTCUpdated ago By Ahmed Aboulenein U.S. Food and Drug Administration Commissioner Marty Makary speaks during a press conference at the Department of Health and Human Services in Washington, D.C., U.S., November 10, 2025.... Purchase Licensing Rights, opens new tab Read ...

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]

We set up a state-f funded newborn screening program to offer children whole [music] genome sequencing at birth free of charge. So, it's an issue. >> Any parent who wants to put their child into this.>> Yes, it's [music] an opt-in program. So, it involves their consent. >> When you think about Drew, is [music] he forever four years old in your mind or >> Oh, that's a great question.>> [music] >> This is the path. I'm Becky Quick. Thank you for coming back.[music] >> In my 25 years at CNBC reporting on the s ...

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]

Market Dynamics & Investment Opportunities - The pharmaceutical complex experienced a 10% increase in two days last week, a phenomenon unseen in over 20 years [3] - A confluence of factors, including potential tariff resolutions, MFN (most favored nation) drug pricing considerations, and renewed pharma M&A activity, are contributing to a changing investment landscape in the sector [3][4] - Lower interest rates are creating a favorable environment for biotech and small-to-mid-cap (smidcap) companies, enabling them to raise capital and grow [7][8] - New biotech companies are emerging as leaders, demonstrating strong growth and successful drug launches, challenging the established leaders of the past decade [6] M&A Activity - Increased M&A activity, particularly involving pre-commercial companies, signals positive momentum in the biotech sector [4] - Limited M&A activity in recent years has allowed strong new biotech companies to emerge as leaders [5] Company Specific Analysis (UniQure) - UniQure's stock price surged 200% in one month following the release of three-year data from their gene therapy program for Huntington's disease [9] - UniQure's gene therapy program demonstrated a 75% reduction in the effect of the damaged gene in Huntington's disease patients [11] - The potential market for UniQure's Huntington's disease treatment is estimated to be multi-billion dollars, making the company's valuation attractive [12]

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. ...