Avidity initiating global Phase 3 HARBOR™ study for delpacibart etedesiran this quarter Delpacibart etedesiran data from MARINA-OLE™ showed reversal of disease progression in multiple functional measures in DM1 compared to END-DM1 natural history data SAN DIEGO, May...
“$4.25 million!!!” That’s the email Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research (CBER), said he received (give or take an exclamation point) from Commissioner Robert Califf when Orchard Therapeutics revealed the price tag of its...
DURHAM, N.C.–(BUSINESS WIRE)– Precision BioSciences, Inc., (Nasdaq: DTIL), an advanced gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for sophisticated gene edits, including gene insertion,...
In a notable advancement for hemophilia treatment, Pfizer Inc. (NYSE: PFE) has received U.S. Food and Drug Administration approval for BEQVEZ™ (fidanacogene elaparvovec-dzkt), a one-time gene therapy aimed at adults suffering from moderate to severe hemophilia B. This...
The FDA’s Center for Biologics Evaluation and Research is planning to release some crucial guidance soon for CRISPR and gene therapy developers on platform technologies, accelerated approvals for rare diseases, and potentially in the “not-too-distant future,” a...