Pharmaceuticals, Biotechnology and Life Sciences
—Low Dose Showed Positive Increases in Cardiac Protein Expression—Two Patients With >50% by IHC, One at Month 9 and One…
Rocket Pharmaceuticals has received clearance from the U.S. Food and Drug Administration (FDA) for the Company’s Investigational New Drug (IND) application for RP-L401, its, lentiviral vector (LVV)-based gene therapy for the treatment of Infantile Malignant Osteopetrosis (IMO), a rare, severe monogenic bone resorption disorder.
Rocket Pharmaceuticals has reported a strong balance sheet with $275.9 million in cash, capitalized into 2022.
NEW YORK–(BUSINESS WIRE)–Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies…
Rocket Pharmaceuticals said that two critical validations for an FA gene therapy product are: 1) stem cell engraftment in the…
NEW YORK–(BUSINESS WIRE)–Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a leading U.S.-based multi-platform clinical-stage gene therapy company, today announces the clearance…
–Rocket’s Largest Lentiviral Pipeline Opportunity with an Estimated Prevalence of 3,000 to 8,000 Patients in the U.S. and EU– –Phase…
– First Patient Dosed in RP-A501 Trial for Danon Disease – – Multi-platform Gene Therapy Pipeline Advances with Four Rocket-sponsored…