Cambridge, Mass.-based bluebird bio and Bagsvaerd, Denmark-based Novo Nordisk agreed to develop next-generation genome editing therapies for genetic diseases, including hemophilia.
Pfizer Inc. is investing $500 million to expand a manufacturing facility in Sanford, North Carolina, that plays a central role in the company’s efforts to become a major player in gene therapy.
An unnamed Novartis executive sold 925,400 Swiss francs ($946,000) worth of shares less than three weeks before the U.S. Food and Drug Administration announced data from tests of the company’s gene therapy Zolgensma had been manipulated.
Days after the U.S. FDA revealed data manipulation was involved in the early studies of Novartis’ multi-million dollar gene therapy for SMA, U.S. Senate members demanded the regulatory agency take action against AveXis, the company that developed Zolgensma.
Sarepta Therapeutics Inc. was informed that an adverse event report was erroneously submitted to the U.S. health regulator regarding an ongoing study of the company’s gene therapy for Duchenne muscular dystrophy (DMD).
The U.S. Food and Drug Administration revealed that data manipulation was involved in the preclinical process for Novartis’ gene therapy Zolgensma as a one-time treatment for spinal muscular atrophy, but suggests that the therapy remain on the market.
Research from three clinical trials supports that Sarepta Therapeutics’ Exondys 51 slows respiratory decline in DMD.
Pfizer’s IV Revatio, when added to inhaled nitric oxide, failed to meet the primary efficacy endpoint in treating newborns with persistent pulmonary hypertension in a Phase III study.
The U.S. FDA approved Symdeko (tezacaftor/ivacaftor and ivacaftor) for use in children with cystic fibrosis ages 6 through 11 years who have two copies of the F508del-CFTR mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator gene that is responsive to the Vertex Pharmaceuticals Inc. medicine.
Orchard Therapeutics Licenses Potentially Life-Changing MPS-I Gene Therapy Program
ADA Severe Combined Immune Deficiency (ADA-SCID), Business, Gene Therapy, Gene Transfer Technologies, Hematopoietic Stem Cell Transplantation (HSCT), Hurler syndrome (MPS I), Licensing, Neurometabolic Disorders, Stem Cell TherapyOrchard Therapeutics secured an exclusive license for an ex vivo autologous hematopoietic stem cell gene therapy program for the treatment of MPS-I developed by SR-Tiget.
