J.P. Morgan analyst Eric Joseph maintained a Buy rating on Rocket Pharmaceuticals (RCKT – Research Report) yesterday and set a price ...

RP-L102, which the firm designed to treat a form of disease caused by FANCA mutations, is already under review with the European Medicines Agency.

TD Cowen analyst Tyler Van Buren has maintained their bullish stance on RCKT stock, giving a Buy rating today. Tyler Van Buren has given his ...

Inc. ("bluebird bio" or the "Company") today announced that it partially adjourned its annual meeting of stockholders ("Annual Meeting") on Wednesday, November 6, 2024, to allow the Company to solicit ...

RCCS San Raffaele Scientific Institute in Milan researchers have discovered that hematopoietic stem cells (HSCs) adapt their lineage commitment during gene therapy based on the underlying genetic ...

Adeno-associated viral vectors (AAV) have emerged as key players in this growth trajectory, projected to capture a ...

In vivo cell therapy is a new way of delivering cell therapy, but the actual therapies don’t need to be new. In theory, ...

Reporting in the peer-reviewed journal Human Gene Therapy, researchers used ex vivo lentiviral gene therapy to treat mucopolysaccharidosis IVA (MPS ...

Without dystrophin, muscle becomes fragile and easily degenerates. A new single-dose intravenous gene therapy, delandistrogene moxeparvovec, creates “micro-dystrophin” — a scaled-down version of the ...

Researchers at the Karolinska Institute continue to investigate whether gene therapy can improve vision in people with Bothnia dystrophy, a form of hereditary blindness, prevalent in the region ...

CHOP Hematology Chief has been elected to the prestigious organization ...

One of two new studies found most patients doing well six years after treatment. Another study found blood cancers in 17% of patients given the therapy using a slightly different protocol. Cerebral ...