Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type II

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Research area:

Rare disease

Disease:

Mucopolysaccharidosis type II

Intervention:

Therapeutics – ATMP

Phase:

Phase I/II

Intervention

Therapeutics – ATMP

Funding awarded to

University of Manchester

Public registration details (where available)

Planned trial start and expected end date

June 2023 to September 2027