Cell and Gene Therapy
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Dispatch seeks to address two main challenges of immunotherapies in solid tumors: the lack of a target and the immunosuppressive tumor environment.
The voluntary pauses follow two patient deaths associated with the Duchenne muscular dystrophy gene therapy.
Sarepta Therapeutics’ stock has dropped precipitously as questions swirl around the safety of its gene therapies. Meanwhile, the Duchenne patient community fears losing access to Elevidys while the regulator considers more drastic action.
After initially refusing to suspend Elevidys distribution after two deaths, Sarepta has now given in to the FDA’s request, noting the need to maintain a good working relationship with the regulator.
In light of recent patient deaths, the FDA has also revoked its platform designation for Sarepta’s AAVrh74 technology. The designation, granted last month, was the first of its kind to be announced publicly.
Amid a season of regulatory and scientific advances, experts reveal a culture of data hoarding among cell and gene therapy developers that is reinforcing fragmentation, stalling innovation and delaying access to treatments.
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.