Cell therapy
While BMO Capital Markets said that zimislecel is “highly encouraging” for type 1 diabetes, questions regarding its target population and Vertex’s execution hang over the cell therapy’s commercial potential.
The well-respected director of the FDA’s cell and gene therapy office was seen as a stabilizing and trustworthy voice inside the quickly reshaping FDA, especially since the late-March exit of CBER Director Peter Marks.
Analysts at Truist Securities called J&J’s CAR T readout “compelling,” noting that the efficacy figures could position the cell therapy as a formidable competitor to the current standard of care, Gilead’s Yescarta.
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.
J&J has a multi-year head start, but Gilead believes it can win market share by delivering a drug with better safety and at least as good efficacy.
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.
After warnings that the dragged-out process was putting the cell therapy company at risk of bankruptcy, bluebird bio now has a new deal to offer shareholders.
It’s another wild twist in the story of Galapagos, a company that has been around for more than 25 years but has yet to get a therapy approved.
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