Pfizer Inc. won U.S. approval for its first gene therapy, expanding into a promising field of medicine that has proved to be a challenging business. The treatment, called Beqvez, gained clearance ...
An iEcure spokesperson said the company anticipates dosing the first patient its phase 1 study of the experimental ...
Pharma has over $500B in capital for deals, analysts note — with companies like Novo Nordisk, Eli Lilly, and Merck continuing to be open to early-stage deals: ...
A young boy with Duchenne muscular dystrophy (DMD) participating in Pfizer’s phase 2 gene therapy trial has died, the pharma shared in a May 7 ...
A PDUFA target action date of November 13, 2024 has been set for the application. The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for ...
Hello, everyone, and welcome to the middle of the week. Congratulations on making it this far, because it is an accomplishment, after all. The next step, of course, is to somehow forge ahead.
Launched in 2021, the public-private consortium on Wednesday updated ASGCT attendees on its efforts to bring adeno-associated ...
Pfizer has paused enrollment in a Phase III trial assessing its Duchenne muscular dystrophy (DMD) gene therapy candidate ...
In an important step toward more effective gene therapies for brain diseases, researchers from the Broad Institute of MIT and Harvard have engineered a gene-delivery vehicle that uses a human protein ...
PTC Therapeutics has won U.S. Food and Drug Administration priority review for its application seeking approval of its Upstaza gene therapy for the treatment of AADC deficiency, a rare genetic ...
An early-stage clinical experiment published in the New England Journal of Medicine found that gene therapy can improve hereditary blindness. Researchers from the Children's Hospital of Philadelphia ...
WARREN, N.J. - PTC Therapeutics, Inc. (NASDAQ: NASDAQ:PTCT) announced today that its Biologics License Application (BLA) for Upstaza™ (eladocagene exuparvovec) has been accepted by the U.S. Food ...