Clinical Trial Update: Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant [Phase 1, Phase 2]

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Summary

Clinical trial update on a gene therapy using modified autologous hematopoietic stem cells to treat Mucopolysaccharidosis type I, Hurler syndrome; the Phase 1/2…

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Diseases / indications

• Mucopolysaccharidosis Type Hurler Syndrome

Drugs mentioned

• Gene therapy with modified autologous hematopoietic stem cells

Snippet

Sponsor: Orchard Therapeutics | Conditions: Mucopolysaccharidosis IH | Phase 1, Phase 2 | Status: ACTIVE_NOT_RECRUITING