Cell and Gene Therapy
THE LATEST
On the heels of a Phase III flop for Pfizer’s Duchenne muscular dystrophy gene therapy candidate, the FDA has green lighted the expanded use of Sarepta Therapeutics’ Elevidys.
The next six months for the FDA are primed to be as groundbreaking as the first six, with Eli Lilly’s donanemab and Lykos Therapeutics’ MDMA-assisted PTSD therapy on the docket, among others.
The FDA is facing four big target action dates in the final week of June, including one label expansion for a bispecific antibody and another for an investigational gene therapy.
The plethora of genes involved in obesity presents an intriguing opportunity for both gene silencing and ex vivo gene therapy approaches.
Results of a large Stanford Medicine study, published Wednesday in The New England Journal of Medicine, finds CAR-T therapies carry a low risk of secondary malignancies not related to the T cells.
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.