Drugs / BIDRIDISTROGENE XEBOPARVOVEC
Trials 3
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT05876780 | Dec 2022 → Aug 2028 expected | limb-girdle muscular dystrophy | Sarepta Therapeutics, Inc. | Active not recruiting | No outcome recorded |
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT03652259 | Oct 2018 → Jan 2025 | autosomal recessive limb-girdle muscular dystrophy type 2E | Sarepta Therapeutics, Inc. | Terminated | No outcome recorded Stop: Business |
| Phase 31 trial | ||||||
| Phase 3 | NCT06246513 | Jan 2024 → Mar 2025 overdue | limb-girdle muscular dystrophy | Sarepta Therapeutics, Inc. | Active not recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 7
| Date | Issuer | Release |
|---|---|---|
| 2025-06-04 | Sarepta Therapeutics, Inc. | Regulatory U.S. FDA Grants Platform Technology Designation to the Viral Vector Used in SRP-9003, Sarepta’s Investigational Gene Therapy for the Treatment of Limb Girdle Muscular Dystrophy Type 2E/R4 sarepta.com ↗ |
| 2021-03-18 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics’ Investigational Gene Therapy SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E Shows Sustained Expression and Functional Improvements 2 Years After Administration sarepta.com ↗
In the first look at expression data from biopsies taken two years after a single administration of SRP-9003, results found sustained protein expression in muscle tissue. |
| 2021-03-15 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics to Present Results from its Gene Therapy and RNA Platforms at the 2021 Annual MDA Clinical and Scientific Conference sarepta.com ↗
New, two- and one-year data including expression data from muscle biopsies taken two years post-treatment, from Study 9003-101 of SRP-9003, an investigational gene therapy for the treatment of limb-girdle muscular dystrophy (LGMD) type 2E/R4; |
| 2020-09-28 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics Investigational Gene Therapy SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E Shows Sustained Functional Improvements 18-months After Administration sarepta.com ↗
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced positive results from the ongoing study of SRP-9003 (rAAVrh74.MHCK7.hSGCB), the Company’s investigational gene therapy for limb-girdle muscular dystrophy Type 2E (LGMD2E). |
| 2020-06-08 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics Announces Positive Expression and Functional Data From the SRP-9003 Gene Therapy Trial to Treat Limb-Girdle Muscular Dystrophy Type 2E sarepta.com ↗
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced positive results from a study of SRP-9003, its investigational gene therapy for limb-girdle muscular dystrophy Type 2E (LGMD2E). |
| 2019-11-07 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics Announces Third Quarter 2019 Financial Results and Recent Corporate Developments sarepta.com ↗
We also announced positive nine-month functional results from a cohort of three limb-girdle muscular dystrophy Type 2E clinical trial participants who received our investigational gene therapy candidate, SRP-9003. |
| 2019-10-04 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics Announces Positive Functional Results from the SRP-9003 (MYO-101) Gene Therapy Trial to Treat Limb-Girdle Muscular Dystrophy Type 2E, or Beta-Sarcoglycanopathy sarepta.com ↗
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced the nine-month functional results from three Limb-girdle muscular dystrophy Type 2E (LGMD2E) clinical trial participants who received SRP-9003. |
All press releases naming this drug 12 releases
Evidence & citations 7 cited values
Every value below carries the sentence it was read from. 5 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | BIDRIDISTROGENE XEBOPARVOVEC | ChEMBL registry synonym — accepted as the source's own label CHEMBL4650240 ↗ |
| Known as | Scaavrh74.mhck7.hsgcb | ChEMBL registry synonym — accepted as the source's own label CHEMBL4650240 ↗ |
| Known as | SRP-9003 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT06246513 ↗ |
| Action | Restore | “We developed a gene therapy approach based on functional replacement of the deficient SCB protein.” PMID 38177855 ↗ Jan 2024 |
| Modality | Gene therapy (AAV / viral vector) | “Here we report interim results from a first-in-human, open-label, nonrandomized, phase 1/2 trial evaluating the safety and efficacy of bidridistrogene xeboparvovec, an...” PMID 38177855 ↗ Jan 2024 |
| Route | Intravenous | “Patients aged 4-15 years with confirmed SGCB mutations at both alleles received one intravenous infusion of either 1.85 × 1013 vector genome copies kg-1 (Cohort 1, n = 3) or...” PMID 38177855 ↗ Jan 2024 |
| Target | SGCB | “an adeno-associated virus-based gene therapy containing a codon-optimized, full-length human SGCB transgene” PMID 38177855 ↗ Jan 2024 |