PGN-EDODM1, an experimental DM1 therapy from Pepgen, showed biomarker activity and splicing correction in an early clinical trial.

ARTHEx Biotech, a clinical-stage biotechnology company advancing RNA-based therapeutics for neuromuscular disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast ...

Ongoing; DT-818 (DM1) Dosing in Patients Expected in the First Half of 2026 Cash and Securities of $219.8 Million as of Year-End Supports Ongoing Clinical Execution CARLSBAD, Calif., March 09, 2026 ...

Dyne has aligned with the U.S. Food and Drug Administration (FDA) on the HARMONIA Phase 3 trial design and protocol. HARMONIA is intended to serve as a confirmatory trial to support conversion of ...

Frontage Laboratories completes acquisition of Teddy Lab to strengthen global central laboratory capabilities: Exton, Pennsylvania Saturday, March 7, 2026, 18:00 Hrs [IST] Frontag ...

Patient dosing in the FREEDOM2 10 mg/kg cohort is proceeding in Canada and the UK, with data expected in 2H 2026 –– FREEDOM2 trial open in South Korea, Australia, and New Zealand –– Well-funded with ...

U.S. FDA places FREEDOM2 trial on partial clinical hold related to preclinical pharmacology and toxicology -- Received regulatory clearance to initiate FREEDOM2 in South Korea, Australia, and New ...

In this phase 1-2 randomized controlled trial involving adults with myotonic dystrophy type 1, del-desiran was well-tolerated in most patients 2. The treatment was also associated with decreased ...

As part of its talent strategy, Sai Life Sciences has launched a global alumni engagement platform and is strengthening learning, leadership development, and internal mobility programs to support long ...

Del-desiran effectively delivered siRNA to muscle, resulting in approximately 40% mean reduction in DMPK mRNA and amelioration of missplicingTreatment ...

Delpacibart etedesiran led mostly to mild to moderate adverse events in a phase I/II trial of myotonic dystrophy type 1, but two serious events occurred. The antibody-oligonucleotide conjugate reduced ...