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 ...
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: ...
Launched in 2021, the public-private consortium on Wednesday updated ASGCT attendees on its efforts to bring adeno-associated ...
The FDA approved both sickle cell treatments at the same time. Bluebird’s gene therapy has been seen as an underdog in this matchup because it has a higher price tag and more safety warnings .
Pfizer has paused enrollment in a Phase III trial assessing its Duchenne muscular dystrophy (DMD) gene therapy candidate ...
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.
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 ...
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 letter (PDF) cited by nonprofit Parent Project ...
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 ...
Pfizer has paused its trial of a drug for a rare muscle-wasting disease after a child died suddenly. The boy, who was between two and four years old, suffered cardiac arrest after receiving the ...
While these technologies are now a therapeutic reality, the ASGCT 2024 annual meeting this week was a reminder of just how ...