Gene therapy
The company’s intein-based technology is initially aimed at Stargardt disease, a type of macular degeneration.
The layoffs will heavily affect Vertex’s operations in Rhode Island, where the biotech will consolidate three facilities into one.
HHS Secretary Robert F. Kennedy Jr. made waves this week, firing the remaining members of the CDC’s Advisory Committee on Immunization Practices; Metsera’s amylin drug produced weight loss of 8.4% at 36 days; and FDA leaders gathered last week to discuss the future of cell and gene therapy, a sector that has been in turmoil since the ousting of CBER Chief Peter Marks.
Gene therapies have ridden investor mania to huge valuations but commercialization challenges have pushed market caps to the floor. At a roundtable last week, FDA leaders promised faster approvals and broad support to the industry.
In a roundtable event on Thursday, HHS Secretary Robert F. Kennedy Jr. said his office will work to eliminate barriers that keep cell and gene therapies from the market.
Interim results from a small group of children in a Phase I/II trial are essentially in line with that of Elevidys, according to BMO Capital Markets analysts.
The Platform Technology Designation, which predates the current FDA leadership, is designed to streamline the drug development and review process, particularly for rare diseases.
While an adverse event reported in Intellia’s gene therapy trial was a “non-concern” for analysts, it follows a handful of patient deaths in other trials for the modality and sent the company’s stock tumbling in pre-market trading.
Acute systemic infection caused the patient to develop fatal capillary leak syndrome, highlighting the unpredictability of gene therapies and potentially challenging investment in the space, analysts say.
Taking center stage at the American Society of Gene and Cell Therapy meeting was the first-ever reported case of a personalized in vivo CRISPR editing therapy, which substantially eased the symptom burden in an infant.
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