This week in InnovationRx focuses on Sarepta’s clash with the FDA, Moderna’s utilization of quantum computers, a new top drug regulator at the FDA, ARCH Venture’s latest biotech company, and more. To receive this newsletter in your inbox, subscribe here.
Sarepta Therapeutics CEO Douglas Ingram
Bloomberg Finance
Usually when the FDA asks a drugmaker to halt distribution of a treatment, the company complies.
Last week, in a remarkable showdown, Sarepta Therapeutics refused to stop distributing Elevidys, its therapy for Duchenne muscular dystrophy, as requested by the FDA. However, on Monday, Sarepta decided to pause all shipments of Elevidys by Tuesday evening. This decision came after Children’s Hospital Los Angeles suspended the use of the drug due to regulatory uncertainties.
Elevidys, a gene therapy for Duchenne muscular dystrophy, was first approved in 2023 in a controversial decision made unilaterally by Peter Marks, a top FDA official, despite concerns from his staff.
Following the FDA’s request to halt distribution, Sarepta initially resisted but eventually complied. The company is working on an enhanced immune-suppressing protocol to address potential liver toxicity issues with Elevidys.
The standoff between Sarepta and the FDA comes at a time of slowed investment in gene therapy and internal challenges at Sarepta. The company’s stock has declined significantly in recent days.
It remains uncertain how long the distribution pause will last as Sarepta navigates the path back to market compliance.
Exploring Quantum Computing at Moderna
Moderna
Messenger RNA (mRNA) technology has shown great promise for future vaccines and therapies, especially during the COVID-19 pandemic. One of the challenges in developing new treatments is optimizing mRNA for both durability and effectiveness.
Moderna, renowned for its COVID vaccines, is leveraging quantum computing to address this challenge. By partnering with IBM, Moderna utilizes quantum hardware to optimize mRNA structures, a task that conventional computers struggle with due to the complexity of mRNA combinations.
Quantum computers offer a unique advantage in solving large-scale optimization problems, enabling Moderna to explore new possibilities for mRNA therapies beyond traditional approaches.
Biotech and Pharma Updates
Dispatch Bio, a biotech startup, aims to revolutionize CAR-T cell therapy for solid tumors by developing a novel immunotherapy approach. The company recently emerged from stealth with significant funding to advance its innovative cancer treatment.
Additionally, AstraZeneca plans to invest $50 billion in U.S. drug development by 2030 amidst evolving trade policies.
Digital Health and AI Advancements
Slingshot AI, a mental healthcare startup, introduced an AI-powered chatbot, Ash, to provide support for individuals facing mental health challenges. The company also secured significant funding to enhance its AI-driven mental health solutions.
Moreover, Aidoc, a company specializing in AI algorithms for radiologists, raised $150 million to further develop its healthcare technology.
Public Health and Hospital Updates
The FDA appointed George Tidmarsh, a seasoned pediatric oncologist and pharma executive, as the new director of its Center for Drug Evaluation and Research. Tidmarsh brings a wealth of experience in drug development and regulation to his new role at the FDA.
Notable Articles
Explore how a push for increased organ donation led to controversial practices in organ transplant procedures.
Learn about wellness startup Truemed’s innovative approach to using tax-free funds for healthcare expenses, including purchases of high-end fitness equipment.
Discover the legal battle between Steward Health and its former CEO over alleged insider transactions that led to the healthcare system’s financial troubles.
Read about advancements in delivering vaccines using unconventional methods, such as dental floss, showing promise in animal testing.
Explore the intersection of AI and cancer research through a substantial donation from former Citigroup chair Sandy Weill.
More from Forbes
Check out how Vibe Coding propelled a Swedish AI unicorn to become the fastest-growing software startup.
Learn about JPMorgan’s new fees for fintechs accessing data and its implications for the financial industry.
Discover the unique approach to office snacks in response to Congressional policies.
