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 ...

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 ...

Rocket Pharmaceuticals, Inc. , a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that ...

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.

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

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 ...

That therapy uses an adeno-associated virus (AAV) as a vector rather than a lentivirus, a vector type that features in approved gene therapies such as Roche's Luxturna and Novartis' Zolgensma.

Researchers uncover a link between impaired glutathione–ascorbate metabolism and early dopamine increases in Huntington's ...

These preclinical findings could lead ... Single-Dose Gene Therapy Is Potentially Life-Changing for Adults With Hemophilia B Sep. 25, 2024 — Adults with hemophilia B saw their number of bleeding ...

Spark Therapeutics is on the cusp of gaining the first ever approval for a gene therapy from the FDA, a groundbreaking treatment which restores vision to children and adults with a rare inherited ...