Gene therapy
Three fatalities occurring recently in China 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.
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.
In this episode of Denatured, you’ll hear from Jack Crawford, CEO of Demeetra, and Magnus Gustavsson, chief commercial officer at NorthX Biologics. We unpack the evolution of cell line development — CHO cells, targeted integration, transposases and the collaboration models speeding biologics from sequence to GMP.
The FDA approved the expansion of Casgevy, which had previously been greenlit for patients 12 and up, into a younger pediatric population under the agency’s Commissioner’s National Priority Voucher program.
Teams at facilities being developed by Eli Lilly, Regeneron and other companies will receive early technical guidance and additional perks from the FDA.
A few short days after announcing an FDA pivot on a separate asset, REGENXBIO is planning to test the agency’s apparent newfound rare disease outlook on another late-stage gene therapy.
If cell and gene therapy makers are going to achieve their mission to improve patients’ lives, the industry must come together to share information across stakeholders, from regulators to manufacturers to payers.
Instead of using viral vectors, SonoThera’s genetic medicines are delivered through an ultrasound-mediated technology that could help sidestep key safety issues with conventional delivery methods.
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