Cell and Gene Therapy
THE LATEST
After treatment with a CD19 CAR-T therapy, patients with systemic lupus erythematosus, idiopathic inflammatory myositis or systemic sclerosis achieved long-lasting remission, according to results published in the NEJM.
The regulator has granted a priority review of the efficacy supplement for Sarepta Therapeutics’ gene therapy Elevidys with a target decision date of June 21, 2024.
Kelonia Therapeutics’ in vivo gene placement system is being tapped to help Astellas Pharma expand its portfolio of in vivo CAR-T cell therapies for cancer.
Citing the need for more time to review additional Chemistry, Manufacturing and Controls information, the FDA has extended its target action date for Rocket Pharmaceuticals’ investigational gene therapy by three months.
With last year’s approval of Vertex and CRISPR’s Casgevy, it’s the start of a new era of gene editing. But there are still challenges we must face.
Shares of preclinical genetic medicines company Metagenomi tanked more than 30% on Friday afternoon in a disappointing debut for its initial public offering, bucking the trend of positive IPOs so far this year.
Investors drove up the price of Kyverna Therapeutics’ stock by 59% in its initial public offering on Thursday afternoon, the first day of trading, reaching a peak per-share price of $35.01.
BioNTech will pay $50 million in cash and purchase $200 million of Autolus Therapeutics’ shares to progress the companies’ respective CAR-T candidates to commercialization.
On Thursday, Kyverna Therapeutics is debuting on the Nasdaq with an upsized initial public offering which the biotech will use to support its pipeline of anti-CD19 CAR T therapy candidates.
Under the FDA’s compassionate use program, an eyedrop formulation of Krystal Biotech’s Vyjuvek restored the vision of a teenager with the rare genetic disease dystrophic epidermolysis bullosa.