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AOC 1001 (del-desiran)
AOC 1001 is an antisense oligonucleotide conjugate that targets and reduces expression of a disease-causing gene through a tissue-directed delivery approach.
AOC 1001 is an antisense oligonucleotide conjugate that targets and reduces expression of a disease-causing gene through a tissue-directed delivery approach. Used for Desmin-related myopathy (DRM).
At a glance
| Generic name | AOC 1001 (del-desiran) |
|---|---|
| Sponsor | Avidity Biosciences, Inc. |
| Drug class | Antisense oligonucleotide conjugate |
| Modality | Small molecule |
| Therapeutic area | Rare genetic disease |
| Phase | Phase 3 |
Mechanism of action
Del-desiran is designed to selectively deliver antisense oligonucleotides to target tissues using Avidity's AOC (Antisense Oligonucleotide Conjugate) platform. The conjugate binds to a tissue-specific receptor to facilitate cellular uptake and subsequent knockdown of the target gene expression, reducing production of the pathogenic protein.
Approved indications
- Desmin-related myopathy (DRM)
Common side effects
Key clinical trials
- Global Open-Label Extension Study of Del-desiran for the Treatment of DM1 (PHASE3)
- Global Study of Del-desiran for the Treatment of DM1 (PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- AOC 1001 (del-desiran) CI brief — competitive landscape report
- AOC 1001 (del-desiran) updates RSS · CI watch RSS
- Avidity Biosciences, Inc. portfolio CI