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Avidity Biosciences, Inc.
Traitements, essais et publications liés.
Traitements16programmes
Essais9liés
Publications4liées
SourceDBlocale
Traitements
16| Molécule | Indication / population | Phase | Objectif | Pays | Résultat |
|---|---|---|---|---|---|
| AOC 1001AOC 1001-CS2 (MARINA-OLE) (NCT05479981) is a Phase 2 extension of the AOC 1001-CS1 (MARINA) (NCT05027269) study to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients | Myopathies | Phase 2 | À vérifier | United States | À vérifier |
| AOC 1001 (del-desiran)A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1 | Myopathies | Phase 3 | À vérifier | United States, Canada, Denmark, France, Germany, Italy, Japan, Netherlands, … | À vérifier |
| AOC 1020A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Participants with Facioscapulohumeral Muscular Dystrophy (FSHD) | Myopathies | Phase 2 | À vérifier | United States, Canada, United Kingdom | À vérifier |
| AOC 1044A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1044 for the treatment of Duchenne Muscular Dystrophy (DMD) with Gene Mutations Amenable to Exon 44 Skipping | Myopathies | Phase 3 | À vérifier | Belgium, France, Germany, Italy, Spain, United Kingdom | À vérifier |
| AOC-1020A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD) | Myopathies | Phase 3 | À vérifier | United States, Canada, Denmark, France, Germany, Italy, Japan, Netherlands, … | À vérifier |
| Del-desiran (AOC 1001)A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1 | Myopathies | Phase 3 | À vérifier | United States, Canada, Denmark, France, Germany, Italy, Japan, Netherlands, … | À vérifier |
| delpacibart zotadirsenThe purpose of this Managed Access Program is to allow access to delpacibart zotadirsen (AOC 1044) for eligible patients diagnosed with DMD mutations amenable to exon 44 skipping. The patient's Administering Physician should follow the suggested treatment guidelines and comply with all local health authority regulations. | Myopathies | À vérifier | À vérifier | United States | À vérifier |
| delpacibart zotadirsenThe purpose of this Managed Access Program is to allow access to delpacibart zotadirsen (AOC 1044) for eligible patients diagnosed with DMD mutations amenable to exon 44 skipping. The patient's Administering Physician should follow the suggested treatment guidelines and comply with all local health authority regulations. | Myopathies | À vérifier | À vérifier | United States | À vérifier |
| AOC 1044A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1044 for the treatment of Duchenne Muscular Dystrophy (DMD) with Gene Mutations Amenable to Exon 44 Skipping | Myopathies | Phase 3 | À vérifier | Belgium, France, Germany, Italy, Spain, United Kingdom | À vérifier |
| AOC 1044A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1044 for the treatment of Duchenne Muscular Dystrophy (DMD) with Gene Mutations Amenable to Exon 44 Skipping | Myopathies | Phase 2 | À vérifier | United States | À vérifier |
| AOC-1020A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD) | Myopathies | Phase 3 | À vérifier | United States, Canada, Denmark, France, Germany, Italy, Japan, Netherlands, Spain, United Kingdom | À vérifier |
| Del-desiran (AOC 1001)A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1 | Myopathies | Phase 3 | À vérifier | United States, Canada, Denmark, France, Germany, Italy, Japan, Netherlands, Spain, United Kingdom | À vérifier |
| AOC 1020A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Participants with Facioscapulohumeral Muscular Dystrophy (FSHD) | Myopathies | Phase 1/2 | À vérifier | United States, Canada, United Kingdom | À vérifier |
| AOC 1020A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Participants with Facioscapulohumeral Muscular Dystrophy (FSHD) | Myopathies | Phase 2 | À vérifier | United States, Canada, United Kingdom | À vérifier |
| AOC 1001 (del-desiran)A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1 | Myopathies | Phase 3 | À vérifier | United States, Canada, Denmark, France, Germany, Italy, Japan, Netherlands, Spain, United Kingdom | À vérifier |
| AOC 1001AOC 1001-CS2 (MARINA-OLE) (NCT05479981) is a Phase 2 extension of the AOC 1001-CS1 (MARINA) (NCT05027269) study to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients | Myopathies | Phase 2 | À vérifier | United States | À vérifier |
Essais cliniques
9| Molécule | Indication / population | Phase | NCT | Titre | Statut |
|---|---|---|---|---|---|
| AOC 1001 | Myopathies | Phase 2 | NCT05479981 | MARINA-OLE — Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients | COMPLETED |
| AOC 1001 (del-desiran) | Myopathies | Phase 3 | NCT06411288 | HARBOR — Global Study of Del-desiran for the Treatment of DM1 | COMPLETED |
| AOC 1020 | Myopathies | Phase 2 | NCT06547216 | FORTITUDE-OLE — Phase 2 Open-label Extension Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) | ACTIVE_NOT_RECRUITING |
| AOC 1020 | Myopathies | Phase 1/2 | NCT05747924 | FORTITUDE — Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) | COMPLETED |
| Del-desiran (AOC 1001) | Myopathies | Phase 3 | NCT07008469 | HARBOR-OLE — Global Open-Label Extension Study of Del-desiran for the Treatment of DM1 | ENROLLING_BY_INVITATION |
| AOC-1020 | Myopathies | Phase 3 | NCT07038200 | FORTITUDE-3 — A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD | RECRUITING |
| AOC 1044 | Myopathies | Phase 2 | NCT06244082 | EXPLORE44OLE — Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping | ACTIVE_NOT_RECRUITING |
| AOC 1044 | Myopathies | Phase 3 | NCT07587242 | SAFARI44 — A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping | RECRUITING |
| delpacibart zotadirsen | Myopathies | À vérifier | NCT07250737 | Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping | AVAILABLE |
Publications
4| Molécule | Indication / population | Titre | Journal | Date |
|---|---|---|---|---|
| Del-desiran (AOC 1001) | An Antibody-Oligonucleotide Conjugate for Myotonic Dystrophy Type 1. | The New England journal of medicine | ||
| AOC-1020 | Development of a DUX4-targeting antibody oligonucleotide conjugate as a therapy for FSHD. | Nucleic acids research | ||
| AOC 1044 | AOC 1044 induces exon 44 skipping and restores dystrophin protein in preclinical models of Duchenne muscular dystrophy. | Nucleic acids research | ||
| delpacibart zotadirsen | Antibodies to watch in 2026. | mAbs |