Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant

NCT03488394 · clinicaltrials.gov ↗

PHASE1,PHASE2

Phase

ACTIVE_NOT_RECRUITING

Status

8

Enrollment

INDUSTRY

Sponsor class

Conditions

Mucopolysaccharidosis IH

Interventions

GENETIC: Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LV, encoding for the α-L-iduronidase cDNA, in their final formulation medium.

Sponsor

Orchard Therapeutics

Collaborators

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