Cell and Gene Therapy
THE LATEST
With several recent approvals in the space and more on the horizon, BioSpace looks at some of the key decisions and their larger significance both for patients and science.
Brazil’s Ministry of Health and nonprofit Caring Cross announced a collaboration Tuesday aimed at local manufacturing of CAR-T cell and stem cell gene therapies at a much lower cost than Europe and the U.S.
Capstan Therapeutics is looking to move its CAR-T cell candidate forward after securing financing from pharma venture arms and VC firms.
Two days after winning FDA approval, Orchard Therapeutics on Wednesday provided its U.S. launch plans for metachromatic leukodystrophy gene therapy Lenmeldy, which has a wholesale acquisition cost of $4.25 million for the one-time treatment.
The FDA took center stage last week as it approved the first-ever MASH therapy and considered additional approvals for CAR-T therapies, whose safety the agency has been investigating since last year.
To help support the launch of Lyfgenia, bluebird bio on Monday entered into a five-year term loan deal with Hercules Capital that will extend the biotech’s cash runway through the first quarter of 2026.
Orchard Therapeutics on Monday secured the FDA’s first approval for an autologous gene therapy to treat the rare metabolic disease metachromatic leukodystrophy in children.
By votes of 11-0 and 8-3, respectively, an FDA advisory committee Friday deemed the risks of early death for both Johnson & Johnson’s Carvykti and Bristol Myers Squibb’s Abecma acceptable.
Asgard Therapeutics, a Swedish gene therapy biotech, has closed a $32 million Series A round with help from prominent pharma players as it prepares for a 2026 IND.
The FDA approved Bristol Myers Squibb’s Breyanzi for chronic lymphocytic leukemia and small lymphocytic leukemia prior to Friday’s adcomm for the company’s other CAR-T therapy, Abecma.