Sarepta has reported up to 80% knockdown of DMPK RNA in both the nucleus and cytoplasm of patient-derived muscle cells, alongside correction of abnormal RNA splicing in preclinical models. The findings address a central delivery challenge in myotonic dystrophy type 1, while an ongoing Phase 1/2 trial will determine whether the mechanism translates into patient
ARTHEx Biotech reports peer-reviewed preclinical evidence that ATX-01 reaches the brain and improves DM1-related behavior in mice.
Find out how ARTHEx Biotech’s ATX-01 data could reshape RNA-targeted therapy for myotonic dystrophy today!
Human-relevant drug screening is hitting a scale problem. Curi Bio’s Curiverse aims to turn 3D tissue models into industrial workflows.
PepGen’s FREEDOM2 data hint at PGN-EDODM1 activity in DM1, but bigger questions remain. Read what the 5 mg/kg cohort really changes.
Read why Sarepta Therapeutics’ first siRNA data in FSHD1 and DM1 could matter for rare disease drug development and investor sentiment.
NEJM publication of Avidity Biosciences delpacibart etedesiran data raises new questions for DM1 treatment. Discover what this changes next.