Gene therapy
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 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.
Changing how biopharmas package their products, how regulators review new drugs and how mutated genes are fixed could make ultrarare disease treatments possible.
Jefferies analysts called the proxy filing, which is a standard disclosure after a merger agreement, “much more intriguing than normal” given the regulatory turmoil it revealed.
The FDA is assessing the need for “further regulatory action” on Sarepta’s Duchenne muscular dystrophy gene therapy in the aftermath of two patient deaths, though the regulator has not yet specified what action this could be.
Robert F. Kennedy Jr. testified in front of largely combative congresspeople on vaccine policy, his MAHA report and more; the mass leadership exodus at the FDA continues as CDER and CBER shed key staff; Kennedy’s revamped CDC vaccine advisors convene for their first meeting; Novo and Lilly present new data at the American Diabetes Association’s annual meeting; and BioSpace recaps BIO2025.
Isaralgagene civaparvovec is a “potential best-in-class gene therapy for Fabry disease,” according to analysts at H.C. Wainwright. Sangamo plans to use pivotal Phase I/II data to build an accelerated approval case for the asset.
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