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OTL-203 versus allogeneic HSCT for Hurler syndrome
2022-500306-17-00Phase 3Authorised
- Sponsor
- Orchard Therapeutics
- Start
- Apr 2024
- Modality
- Bone Marrow HSCs → Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies, Peripheral Blood HSCs → Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies, Lentiviral Vectors → Retroviral Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Countries
- 4
01
Overview
Phase 3, randomised, open-label trial in mucopolysaccharidosis type I comparing OTL-203 gene therapy arm with Standard-of-care allogeneic HSCT arm (2 arms). Primary endpoint: Event-free survival at Year 2. Planned start Apr 2024; status: authorised.
02
Drugs
OTL-203
03
Conditions
mucopolysaccharidosis type I
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