Drugs / delpacibart etedesiran
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT05027269 | Oct 2021 → Feb 2023 | myotonic dystrophy type 1 | Avidity Biosciences, Inc. | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 1
| Date | Issuer | Release |
|---|---|---|
| 2026-09-08 | Novartis Pharmaceuticals | Results Novartis provides update on delpacibart etedesiran (del-desiran) Phase III HARBOR study for the treatment of myotonic dystrophy type 1 (DM1) novartis.com ↗
Evidence of clinical activity in secondary endpoints and exploratory analyses were observed. |
All press releases naming this drug 1 release
| Date | Issuer | Release |
|---|---|---|
| 2026-09-08 | Novartis Pharmaceuticals | Novartis provides update on delpacibart etedesiran (del-desiran) Phase III HARBOR study for the treatment of myotonic dystrophy type 1 (DM1) novartis.com ↗ |
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | delpacibart etedesiran | “Delpacibart etedesiran (del-desiran [AOC 1001]) is a monoclonal antibody-oligonucleotide conjugate.” PMID 41707138 ↗ Feb 2026 |
| Known as | AOC 1001 | “Delpacibart etedesiran (del-desiran [AOC 1001]) is a monoclonal antibody-oligonucleotide conjugate.” PMID 41707138 ↗ Feb 2026 |
| Known as | del-desiran | “Delpacibart etedesiran (del-desiran [AOC 1001]) is a monoclonal antibody-oligonucleotide conjugate.” PMID 41707138 ↗ Feb 2026 |
| Action | Inhibit | “The percent change in DMPK mRNA levels in muscle-biopsy samples was -46% in the 1-mg group, -44% in the 2-mg group, -37% in the 4-mg group” PMID 41707138 ↗ Feb 2026 |
| Target | DMPK | “the oligonucleotide component targets DMPK mRNA.” PMID 41707138 ↗ Feb 2026 |