First Participant Dosed in AskBio Phase 1/Phase 2 Gene Therapy Trial of AB-1009 for Late-Onset Pompe Disease (LOPD)

AB-1009, an adeno-associated virus (AAV) investigational gene therapy, is being evaluated for the treatment of late-onset ...
The Manila Times

Dyne Therapeutics Reports First Quarter 2026 Financial Results and Recent Business Highlights

Positive pre-BLA meeting completed with FDA for z-rostudirsen in exon 51 DMD; on track for BLA submission in Q2 2026 and potential launch in Q1 2027 - ...
BioPharma Dive

Inhibrx delivers ‘differentiated’ cancer drug data; Fractyl starts diabetes gene therapy trial

Inhibrx, the subject of recent buyout rumors, showed its drug might enhance the effects of Keytruda. Elsewhere, Capricor sued its cell therapy partner and Argenx got a revenue boost.
Armagh I

Hope for Newry DMD warrior and countless others like him as ‘life-changing’ medication finally approved

The family of a Newry boy who fought for access to a new “life-changing” medication have hailed May 8 as a "very, very good ...