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Dyne Therapeutics
Traitements, essais et publications liés.
Traitements8programmes
Essais4liés
Publications3liées
SourceDBlocale
Traitements
8| Molécule | Indication / population | Phase | Objectif | Pays | Résultat |
|---|---|---|---|---|---|
| DYNE-101The primary purpose of the study is to evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with Myotonic Dystrophy Type 1 (DM1). The study consists of 4 periods: A Screening Period (up to 8 weeks), a Placebo-Controlled Period (24 weeks), a Treatment Period (24 weeks) and a Long-Term Extension (LTE) Period (208 weeks) in both multiple-ascending dose (MAD) and dose expansion cohorts. | Myopathies | Phase 1/2 | À vérifier | United States, Australia, France, Germany, Italy, Netherlands, New Zealand, United Kingdom | À vérifier |
| DYNE-251The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The study consists of 3 periods: a multiple-ascending dose (MAD) / placebo-controlled period (24 weeks), an open-label period (24 weeks) and a long-term extension (LTE) period (288 weeks). | Myopathies | Phase 1/2 | À vérifier | United States, Australia, Belgium, Canada, Ireland, Italy, South Korea, Spain, … | À vérifier |
| zeleciment basivarsen (DYNE-101)The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment basivarsen (DYNE-101) for the treatment of myotonic dystrophy 1 (DM1). | Myopathies | Phase 3 | À vérifier | United States, Australia, Belgium, Denmark, France, Germany, Italy, Japan, … | À vérifier |
| Zeleciment Rostudirsen (DYNE-251)The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment rostudirsen (DYNE-251) administered intravenously (IV) every 4 weeks to ambulatory Duchenne muscular dystrophy (DMD) participants, 4 to 18 years of age, with dystrophin mutations amenable to exon 51 skipping. | Myopathies | Phase 3 | À vérifier | United States | À vérifier |
| DYNE-251The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The study consists of 3 periods: a multiple-ascending dose (MAD) / placebo-controlled period (24 weeks), an open-label period (24 weeks) and a long-term extension (LTE) period (288 weeks). | Myopathies | Phase 1/2 | À vérifier | United States, Australia, Belgium, Canada, Ireland, Italy, South Korea, Spain, United Kingdom | À vérifier |
| zeleciment basivarsen (DYNE-101)The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment basivarsen (DYNE-101) for the treatment of myotonic dystrophy 1 (DM1). | Myopathies | Phase 3 | À vérifier | United States, Australia, Belgium, Denmark, France, Germany, Italy, Japan, Netherlands, New Zealand, Spain, United Kingdom | À vérifier |
| Zeleciment Rostudirsen (DYNE-251)The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment rostudirsen (DYNE-251) administered intravenously (IV) every 4 weeks to ambulatory Duchenne muscular dystrophy (DMD) participants, 4 to 18 years of age, with dystrophin mutations amenable to exon 51 skipping. | Myopathies | Phase 3 | À vérifier | United States | À vérifier |
| DYNE-101The primary purpose of the study is to evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with Myotonic Dystrophy Type 1 (DM1). The study consists of 4 periods: A Screening Period (up to 8 weeks), a Placebo-Controlled Period (24 weeks), a Treatment Period (24 weeks) and a Long-Term Extension (LTE) Period (208 weeks) in both multiple-ascending dose (MAD) and dose expansion cohorts. | Myopathies | Phase 1/2 | À vérifier | United States, Australia, France, Germany, Italy, Netherlands, New Zealand, United Kingdom | À vérifier |
Essais cliniques
4| Molécule | Indication / population | Phase | NCT | Titre | Statut |
|---|---|---|---|---|---|
| DYNE-101 | Myopathies | Phase 1/2 | NCT05481879 | ACHIEVE — Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1 | ACTIVE_NOT_RECRUITING |
| Zeleciment Rostudirsen (DYNE-251) | Myopathies | Phase 3 | NCT07608432 | Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO) | RECRUITING |
| zeleciment basivarsen (DYNE-101) | Myopathies | Phase 3 | NCT07486934 | Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1 | RECRUITING |
| DYNE-251 | Myopathies | Phase 1/2 | NCT05524883 | DELIVER — Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping | ACTIVE_NOT_RECRUITING |
Publications
3| Molécule | Indication / population | Titre | Journal | Date |
|---|---|---|---|---|
| DYNE-101 | The myotonic dystrophy type 1 drug development pipeline: 2022 edition. | Drug discovery today | ||
| DYNE-101 | FORCE platform overcomes barriers of oligonucleotide delivery to muscle and corrects myotonic dystrophy features in preclinical models. | Communications medicine | ||
| DYNE-101 | From molecular convergence to clinical divergence: Comparative pathogenic mechanisms and therapeutic trajectories in C9orf72-ALS/FTD and myotonic dystrophy. | Neurobiology of disease |