Cell therapy
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.
As tariffs, HHS workforce cuts and the ouster of CBER Director Peter Marks threaten the “lifeblood” of the cell and gene therapy space, experts express wariness over the unknowns and optimism that Marks’ legacy will carry on.
The cell and gene therapy company is cutting 47 employees and its entire lupus program to focus resources on two CAR Ts. The move follows a reconfiguration last year to move into immunology.
Paul Stoffels left his perch as J&J’s chief scientific officer in 2022 to replace Galapagos’ founding CEO Onno van de Stolpe, inheriting a company that had suffered a series of clinical failures since its 1999 creation.
Already reeling from years of market chaos, the announced departure of CBER chief Peter Marks sent a ripple across biopharma markets.
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