Cell and Gene Therapy
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Following the death of two teenage patients with Duchenne muscular dystrophy following Elevidys treatment, Sarepta Therapeutics adds a black box warning to the gene therapy for acute liver injury and failure and parts with more than a third of employees.
From Wall Street to real estate to a “big, ugly” pharma building, Mayo Venture Partner Audrey Greenberg reflects on a career defined by taking a leap at just the right moment.
The FDA cited manufacturing issues but did not flag problems with Ultragenyx’s data package for UX111, with the biotech noting that the regulator found its neurodevelopmental findings for the gene therapy to be “robust.”
The deal gives AstraZeneca’s rare disease unit Alexion access to specialized capsids developed by the Japanese biotech JCR Pharmaceuticals for use in up to five of Alexion’s gene therapies.
The deal marks an end for CAR T company Cargo Therapeutics, which has been slashing its workforce and cutting programs since the January decision to halt its lead candidate for a certain type of aggressive large B cell lymphoma.
The high court sides with HHS on HIV PrEP drugs; Health Secretary RFK Jr.’s newly appointed CDC vaccine advisors discuss thimerosal in flu vaccines, skip vote on Moderna’s mRNA-based RSV vaccine; FDA removes CAR T guardrails; AbbVie snaps up Capstan for $1.2B to end first half; and psychedelics take off again with data from Compass and Beckley.
Cell and gene therapy leaders say the agency’s decision to remove the Risk Evaluation and Mitigation Strategies that had been attached to approved CAR T cancer therapies reflects “thoughtful consideration of real-world evidence” and “regulatory trust.”
The pivotal trial for Neurogene’s Rett syndrome gene therapy makes use of baseline controls and a rigorous endpoint that could help ensure a broader label for the drug product, if approved, according to analysts.
The all-cash buyout, which gives AbbVie access to Capstan Therapeutics’ in vivo edited CAR T therapy for B cell–mediated autoimmune diseases, adds to a growing sense of momentum in M&A, according to BMO Capital Markets.
Changing how biopharmas package their products, how regulators review new drugs and how mutated genes are fixed could make ultrarare disease treatments possible.