AOC 1001
- Generic Name
- AOC 1001
- Drug Type
- Biotech
- Background
AOC 1001 is an antibody oligonucleotide conjugate (AOC), comprised of a human transferrin receptor 1 (TfR1) targeting, effector function null, humanized IgG1 antibody (AV01mAb) conjugated to one small interfering RNA (siRNA).
Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients
Phase 2
Active, not recruiting
- Conditions
- Musculoskeletal DiseasesDM1Myotonic DystrophyMuscular Disorders, AtrophicMuscular DystrophiesMyotonic DisordersGenetic Diseases, InbornMuscular DiseasesMyotonic Dystrophy 1Neuromuscular Diseases
- Interventions
- Drug: Placebo
- First Posted Date
- 2022-07-29
- Last Posted Date
- 2024-10-14
- Lead Sponsor
- Avidity Biosciences, Inc.
- Target Recruit Count
- 37
- Registration Number
- NCT05479981
- Locations
- 🇺🇸
Stanford University, Palo Alto, California, United States
🇺🇸University of California Los Angeles, Los Angeles, California, United States
🇺🇸Ohio State University, Columbus, Ohio, United States
Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients
Phase 1
Completed
- Conditions
- Dystrophy MyotonicMyotonic DisordersDM1Steinert DiseaseMyotonic Dystrophy 1Myotonic Muscular DystrophyMyotonic Dystrophy Type 1 (DM1)Myotonic Dystrophy
- Interventions
- Drug: Placebo
- First Posted Date
- 2021-08-30
- Last Posted Date
- 2024-02-05
- Lead Sponsor
- Avidity Biosciences, Inc.
- Target Recruit Count
- 38
- Registration Number
- NCT05027269
- Locations
- 🇺🇸
University of California Los Angeles, Los Angeles, California, United States
🇺🇸Stanford University, Palo Alto, California, United States
🇺🇸Kansas University Medical Center, Kansas City, Kansas, United States
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