Fiche société
REGENXBIO Inc.
Traitements, essais et publications liés.
Traitements4programmes
Essais2liés
Publications2liées
SourceDBlocale
Traitements
4| Molécule | Indication / population | Phase | Objectif | Pays | Résultat |
|---|---|---|---|---|---|
| AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMDThis is an observational screening study to evaluate the prevalence of anti-adeno-associated serotype 8 (AAV8) antibodies in participants with Duchenne muscular dystrophy (DMD). | Myopathies | À vérifier | À vérifier | United States | À vérifier |
| RGX-202RGX-202 is a gene therapy designed to deliver a transgene for a novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain. This is a multicenter, open-label dose evaluation clinical study to assess the safety, tolerability, and clinical efficacy of a one-time intravenous (IV) dose of RGX-202 in participants with Duchenne. | Myopathies | Phase 2/3 | Thérapie génique | United States, Canada | À vérifier |
| RGX-202RGX-202 is a gene therapy designed to deliver a transgene for a novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain. This is a multicenter, open-label dose evaluation clinical study to assess the safety, tolerability, and clinical efficacy of a one-time intravenous (IV) dose of RGX-202 in participants with Duchenne. | Myopathies | Phase 2/3 | Thérapie génique | United States, Canada | À vérifier |
| AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMDThis is an observational screening study to evaluate the prevalence of anti-adeno-associated serotype 8 (AAV8) antibodies in participants with Duchenne muscular dystrophy (DMD). | Myopathies | À vérifier | À vérifier | United States | À vérifier |
Essais cliniques
2| Molécule | Indication / population | Phase | NCT | Titre | Statut |
|---|---|---|---|---|---|
| AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD | Myopathies | À vérifier | NCT05683379 | AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD | ACTIVE_NOT_RECRUITING |
| RGX-202 | Myopathies | Phase 2/3 | NCT05693142 | AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD) | ACTIVE_NOT_RECRUITING |
Publications
2| Molécule | Indication / population | Titre | Journal | Date |
|---|---|---|---|---|
| RGX-202 | AAV-mediated gene transfer of a novel microdystrophin ameliorates pathology and enhances muscle function in a mouse model of DMD. | Molecular therapy. Nucleic acids | ||
| RGX-202 | Targeting the creatine transporter SLC6A8: Mechanisms and emerging therapeutic strategies for multiple diseases. | Biochemical pharmacology |