Cell and Gene Therapy
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Pfizer’s investigational Duchenne muscular dystrophy gene therapy, fordadistrogene movaparvovec, failed in a late-stage study to significantly improve motor function in patients versus placebo.
Given their seven-figure price tags, it’s not clear how accessible the would-be cures will be to U.S. patients on public or private insurance.
The American Society of Clinical Oncology annual meeting kicks off today in Chicago, with highly anticipated presentations that include reports on a bispecific antibody, an ADC and a BCMA-targeted CAR-T cell therapy.
While NK cell therapies can potentially avoid the serious side effects sometimes seen with CAR T cell therapies, experts say durability may stall their path to the market.
BioSpace’s Lori Ellis discusses the risks and challenges of cell and gene therapy combination products with DIA speakers James Wabby, AbbVie and Rob Schulz, Suttons Creek, Inc.
Belgian biotech Galapagos is teaming with Blood Centers of America to help deploy its decentralized CAR-T therapy manufacturing platform closer to treatment centers across the U.S.
Following back-to-back approvals in lymphocytic leukemia, Bristol Myers Squibb’s CAR-T therapy Breyanzi on Wednesday won the FDA’s green light for relapsed or refractory follicular lymphoma.
While these technologies are now a therapeutic reality, the ASGCT 2024 annual meeting this week was a reminder of just how far we are from widespread use.
With 15 patients started across its three gene therapies, bluebird bio claims a 138% year-over-year revenue growth and aims to initiate up to around 100 new patients in the current year.
At a Thursday ASGCT 2024 session, CBER Director Peter Marks made the case for a better, “more convergent” global framework on cell and gene therapies, especially for rare diseases.