Cell therapy
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.
The cell engineering company, co-founded by oncologist and writer Siddhartha Mukherjee, does not see a path forward for its pipeline of early-stage cell therapies for two different types of cancer.
As Q1 2025 earnings season continues, tariffs remain top of mind for pharma CEOs and investors. Meanwhile, the American Association for Cancer Research’s annual event kicks off this year’s oncology conference season. Plus, will the FDA become politicized under HHS Secretary RFK Jr.?
The condition, recessive dystrophic epidermolysis bullosa, causes chronic wounds and has an 84% mortality rate by age 40.
PRESS RELEASES