Drugs / OTL-103
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 3 | NCT03837483 | Jan 2019 → Mar 2024 overdue | Wiskott-Aldrich syndrome | Fondazione Telethon | Active not recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 4
| Date | Issuer | Release |
|---|---|---|
| 2021-06-29 | Orchard Therapeutics | Regulatory Orchard Therapeutics Announces Regulatory and Clinical Updates for Lead Gene Therapy Programs orchard-tx.com ↗
These updates follow recent regulatory interactions for multiple programs within the company’s clinical-stage pipeline targeting metachromatic leukodystrophy (MLD), mucopolysaccharidosis type I Hurler syndrome (MPS-IH) and Wiskott-Aldrich syndrome (WAS). |
| 2019-11-06 | Orchard Therapeutics | Results Orchard Therapeutics Reports Third Quarter 2019 Financial Results and Achievement of Key 2019 Milestones for Neurometabolic and Primary Immune Deficiencies Franchises orchard-tx.com ↗
The registrational trial has met its key primary and secondary endpoints (n=8 at three years) with data from an integrated analysis (n=17) reinforcing the treatment benefits of gene therapy and durability of effect in additional patients. |
| 2019-11-06 | Orchard Therapeutics | Results Orchard Therapeutics to Present New Registrational Data of Investigational Gene Therapies at the 61st American Society of Hematology Annual Meeting orchard-tx.com ↗
Registrational Trial for Wiskott-Aldrich Syndrome Met Key Primary and Secondary Endpoints at Three Years |
| 2019-07-29 | Orchard Therapeutics | Regulatory Orchard Therapeutics Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted for OTL-103 for the Treatment of Wiskott-Aldrich Syndrome orchard-tx.com ↗ |
All press releases naming this drug 5 releases
Evidence & citations 2 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | OTL-103 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03837483 ↗ |
| Target | WAS | “Autologous hematopoietic stem cells collected from mobilized peripheral blood transduced ex vivo with a lentiviral vector encoding the WAS cDNA” NCT03837483 ↗ |