Cell and Gene Therapy
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Biopharmas developing CAR T therapies for autoimmune indications face major barriers to maximizing the market. Advances in the provider ecosystem and clinical infrastructure, as well as access and education, will be key to launching at scale.
Setbacks to efforts to use Cellares’ automated platform to make an approved CAR-T cell therapy led BMS to terminate its partnership with the manufacturing specialist.
FDA extends its review of Capricor Therapeutics’ Duchenne muscular dystrophy cell therapy as an unhappy shareholder advocates for change; Merck and Moderna tout positive data for their mRNA-based cancer vaccine; Trump nominates White House aide Heidi Overton as FDA commissioner; and the oral obesity market heats up with rivals Eli Lilly and Novo Nordisk going head-to-head.
The FDA has granted a three-month extension for Capricor Therapeutics’ Duchenne muscular dystrophy hopeful deramiocel in order to review additional data and consider a refined indication. The new target action date for the therapy is November 22.
Three fatalities occurring recently in Chinese clinical trials have spurred two members of U.S. Congress to urge the FDA to enact new policies designed to protect American patients.
The accelerated greenlight for Ultragenyx’s gene therapy for glycogen storage disease has raised analyst expectations for approval of UX111, which the FDA rejected last summer and is currently reviewing for a second time. A decision is expected by mid-September.
Johnson & Johnson secured rights to prizlon-cel in 2023 for $245 million upfront. The asset in June last year showed a 100% response rate as a second-line option in a Phase 1b study for 10 patients with relapsed or refractory large B cell lymphoma.
Capricor Therapeutics’ CEO Linda Marbán believes the FDA is willing to work with the biotech as the Aug. 22 deadline approaches for its Duchenne muscular dystrophy cell therapy deramiocel, with the biotech’s stock climbing 68% on the update.
In November 2025, one patient died after receiving Intellia Therapeutics’ investigational gene silencer for transthyretin amyloidosis.
UniQure is ready to put its Huntington’s disease gene therapy through an advisory committee meeting—even as the broader rare disease space has been put on edge by recent FDA scrutiny of assets by Replimune and Capricor.