Also known as: DMPK
The FDA has placed a partial hold on U.S. enrollment in a Phase 2 trial of del-desiran for myotonic dystrophy type 1, citing preclinical mouse data showing blood pressure drops not seen in humans, following a serious adverse event in earlier testing.
Hyderabad-based CRDMO Sai Life Sciences plans to recruit over 700 scientific, technical, and management professionals during 2026-27, representing a 20% workforce expansion driven by growing global demand for integrated drug discovery and manufacturing services.
Phase 1/2 MARINA trial results published in NEJM show del-desiran achieved approximately 40% reduction in toxic DMPK mRNA in muscle tissue and improvements in myotonia and functional measures in myotonic dystrophy type 1 patients, with acceptable safety profile.
