Drugs / ATA-100
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1 | NCT05224505 | Sep 2022 → Feb 2025 overdue | autosomal recessive limb-girdle muscular dystrophy | Atamyo Therapeutics | Active not recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 13
| Date | Issuer | Release |
|---|---|---|
| 2025-04-03 | Atamyo Therapeutics | Results Atamyo finalise l’étude de choix de dose dans la myopathie des ceintures de type R9 (LGMD-R9) et obtient la désignation de maladie pédiatrique rare de la FDA américaine pour ATA-100 atamyo.com ↗
Efficacité marquée dans la première cohorte ayant reçu la dose 9E12 vg/kg, avec améliorations de tous les critères d’évaluation fonctionnels et histologiques chez tous les patients traités |
| 2025-04-03 | Atamyo Therapeutics | Results Atamyo completes the dose-finding study in Limb-Girdle Muscular Dystrophy Type R9 (LGMD-R9) and obtains US Rare Pediatric Disease Designation for ATA-100 atamyo.com ↗
Satisfactory biodistribution and improvements in histology biomarkers, including αDG glycosylation increases, were observed in patients’ muscle biopsies with both doses. |
| 2024-06-24 | Atamyo Therapeutics | Regulatory Atamyo Therapeutics Obtains US FDA Fast Track Designation for ATA-100, a Gene Therapy in Phase 1/2 Clinical Trials for Limb-Girdle Muscular Dystrophy Type R9 (LGMD-R9) atamyo.com ↗
Atamyo Therapeutics Obtains US FDA Fast Track Designation for ATA-100, a Gene Therapy in Phase 1/2 Clinical Trials for Limb-Girdle Muscular Dystrophy Type R9 (LGMD-R9) |
| 2023-10-27 | Atamyo Therapeutics | Results First clinical results of ATA-100, a Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9), presented at ESGCT atamyo.com ↗
Atamyo Therapeutics , a biotechnology company focused on the development of new-generation gene therapies targeting muscular dystrophies and cardiomyopathies, today announced the presentation of the first clinical results obtained with ATA-100 in the on-going ATA-001 Phase 1b/2b clinical trial. |
| 2023-10-27 | Atamyo Therapeutics | Results Premiers résultats cliniques d’ATA-100, une thérapie génique pour le traitement de la dystrophie musculaire des ceintures de type 2I/R9 (LGMD 2I/R9), présentés au congrès ESGCT atamyo.com ↗
Atamyo Therapeutics , une entreprise biopharmaceutique spécialisée dans le développement de thérapies géniques de nouvelle génération ciblant des maladies neuromusculaires, a annoncé aujourd’hui la présentation des premiers résultats cliniques obtenus avec l’ATA-100 dans l’essai clinique de Phase 1b/2b « ATA-001 » mené actuellement. |
| 2023-09-06 | Atamyo Therapeutics | Regulatory IND for ATA-100, a Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9), cleared to proceed by FDA atamyo.com ↗ |
| 2022-05-16 | Atamyo Therapeutics | Regulatory Atamyo Therapeutics announces Significant Milestones for ATA-100 and ATA-200, its Gene Therapy programs to Treat Limb-Girdle Muscular Dystrophy 2I/R9 and 2C/R5 atamyo.com ↗
A third authorization of a Clinical Trial Application (CTA) in Europe was granted by the French National Medicines Health Agency (ANSM) for ATA-100. |
| 2022-02-24 | Atamyo Therapeutics | Regulatory Atamyo Therapeutics Reaches Significant Regulatory and Financial Milestones for ATA-100, its Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type 2I/R9 atamyo.com ↗
A second authorization of a Clinical Trial Application (CTA) in Europe was granted by the Denmark Medicines Agency (DKMA) for ATA-100. |
| 2022-02-24 | Atamyo Therapeutics | Regulatory Atamyo Therapeutics franchit d’importantes étapes réglementaires et financières pour ATA-100, sa thérapie génique destinée à traiter la dystrophie musculaire des ceintures de type 2I/R9 atamyo.com ↗
Une deuxième autorisation d’une demande d’essai clinique (CTA) en Europe a été délivrée par la Danish Medicines Agency (DKMA) au Danemark pour ATA-100. |
| 2021-12-06 | Atamyo Therapeutics | Regulatory Atamyo Therapeutics obtient une première autorisation réglementaire en Europe pour démarrer un essai clinique pour ATA-100, sa thérapie génique destinée à traiter la dystrophie musculaire des ceintures de type 2I/R9 atamyo.com ↗
Cette autorisation a été délivrée par la Medicines & Healthcare products Regulatory Agency (MHRA) du Royaume-Uni. |
| 2021-12-06 | Atamyo Therapeutics | Regulatory Atamyo Therapeutics Obtains First Regulatory Authorization in Europe to Initiate a Clinical Trial for ATA-100, its Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type 2I/R9 atamyo.com ↗
This authorization was granted by the United Kingdom Medicines & Healthcare products Regulatory Agency (MHRA). |
| 2021-10-26 | Atamyo Therapeutics | Regulatory Atamyo Therapeutics Submits CTA in Europe for ATA-100, Its Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type 2I/R9 atamyo.com ↗ |
| 2021-10-26 | Atamyo Therapeutics | Regulatory Atamyo Therapeutics dépose une demande d’autorisation d’essai clinique en Europe pour ATA-100, sa thérapie génique destinée à traiter la dystrophie musculaire des ceintures de type 2I/R9 atamyo.com ↗ |
All press releases naming this drug 15 releases
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | ATA-100 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05224505 ↗ |
| Known as | GNT0006 | “ATA-100 (Formerly GNT0006) Gene Therapy Trial in Patients With LGMDR9” NCT05224505 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “Adeno-Associated Virus (AAV) vector carrying the human FKRP transgene” NCT05224505 ↗ |
| Route | Intravenous | “Single intravenous infusion on Day 0” NCT05224505 ↗ |