Cell and Gene Therapy

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.

Sarepta’s shares crashed 41% in premarket trading Monday morning to $21.01 after the biotech reported a second death from acute liver failure, a known side effect of adeno-associated virus-based gene therapies.

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.

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.