source
- Novartis' DM1 drug del-desiran misses primary endpoint in Phase 3 HARBOR trialNovartis announced that its experimental therapy del-desiran did not improve muscle function in a pivotal Phase 3 study for myotonic dystrophy type 1 (DM1). The trial, called HARBOR, evaluated the…
- #ERS26: Beam says AATD base editing data hold up after one yearBeam Therapeutics on Tuesday shared additional data from an early-stage trial of its gene therapy candidate in a rare disease that causes lung and liver damage. In six patients in a Phase 1/2 study…Endpoints · 1h ago↗ source