Drugs / rAAVrh74.MCK.GALGT2
rAAVrh74.MCK.GALGT2
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT03333590 | Nov 2017 → Nov 2020 | Duchenne muscular dystrophy | Kevin Flanigan | Completed | No outcome recorded |
| Phase 1/2 | NCT02704325 | Apr 2016 → Jul 2018 | Duchenne muscular dystrophy | Kevin Flanigan | Withdrawn | No outcome recorded |
News releases announcing trial results or a regulatory action · 1
| Date | Issuer | Release |
|---|---|---|
| 2017-11-03 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics and Nationwide Children’s Hospital Announce U.S. Food and Drug Administration (FDA) Clearance of the IND Application for the GALGT2 Gene Therapy Program sarepta.com ↗
Nationwide Children’s Hospital today announced that the Investigational New Drug (IND) application for the GALGT2 gene therapy program was cleared by the FDA. |
All press releases naming this drug 1 release
| Date | Issuer | Release |
|---|---|---|
| 2017-11-03 | Sarepta Therapeutics, Inc. | Sarepta Therapeutics and Nationwide Children’s Hospital Announce U.S. Food and Drug Administration (FDA) Clearance of the IND Application for the GALGT2 Gene Therapy Program sarepta.com ↗ |
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | rAAVrh74.MCK.GALGT2 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03333590 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “Adeno-associated virus serotype rh74 carrying the GALGT2 gene” NCT03333590 ↗ |
| Route | Intramuscular | “Direct intramuscular injection of rAAVrh74.MCK.GALGT2” NCT02704325 ↗ |
| Target | GALGT2 | “evaluated blindly for the expression of the GALGT2 transgene” NCT02704325 ↗ |