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Trial intelligence / Profile preview
OTL-203 gene therapy for Mucopolysaccharidosis type I (Hurler variant)
2024-514870-29-00Phase 2Authorised
- Sponsor
- Orchard Therapeutics
- Start
- Jul 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
- 1
01
Overview
Phase 2, open-label, single-arm trial of OTL-203 in Mucopolysaccharidosis Type I Hurler, sponsored by Orchard Therapeutics. Primary endpoints: Overall survival, Hematologic engraftment by day +45 and 5 more. Planned start Jul 2024; status: authorised.
02
Drugs
OTL-203
03
Conditions
Mucopolysaccharidosis Type I Hurler
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