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Deldesiran

Deldesiran

Drug

Drug Profile

Deldesiran (delpacibart etedesiran, AOC 1001) is an investigational RNA therapeutic candidate for myotonic dystrophy type 1 (DM1). It is being evaluated in ongoing clinical studies and has received multiple FDA expedited development designations. The therapy remains unapproved.

Drug Class: Antibody oligonucleotide conjugate (AOC) RNA therapeutic
Approval Status: Investigational; no approved DM1 therapies
Mechanism of Action: Monoclonal antibody targeting transferrin receptor 1 delivers a conjugated siRNA to reduce disease-related DMPK mRNA

Brand Names

del-desiran
\AOC 1001\
\delpacibart etedesiran\

Indications

\Myotonic dystrophy type 1 (DM1)\

Related News

Avidity's Myotonic Dystrophy Drug Shows Promising Phase 1/2 Trial Results

Mar 26, 2026

Avidity Biosciences announced promising Phase 1/2 trial results for its myotonic dystrophy type 1 drug delpacibart etedesiran, showing 40% reduction in DMPK mRNA and functional improvements. The therapy has received multiple regulatory designations and is now in Phase 3 testing with data expected in late 2026. The company's stock has surged 128% over the past year despite reporting a net loss of approximately $550 million.

FDA Pauses U.S. Enrollment in Myotonic Dystrophy Trial After Serious Adverse Event

Mar 06, 2026

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.

Del-desiran Shows Muscle Delivery and Splicing Improvements in Phase 1/2 DM1 Trial

Feb 19, 2026

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.