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Autologous lentiviral IDS gene therapy in infants with MPS II
ISRCTN12458940Phase 2Active, not recruiting
- Sponsor
- The University of Manchester
- Start
- Mar 2023
- Modality
- Cell Therapies, Gene Therapies
- Countries
- 1
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Overview
Phase 2, open-label, single-arm trial of autologous CD34+ cells transduced with lentiviral IDS.ApoEII in mucopolysaccharidosis type 2, sponsored by The University of Manchester. Primary endpoint: Safety and tolerability of the investigational medicinal product. Started Mar 2023; status: active, not recruiting.
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Drugs
autologous CD34+ cells transduced with lentiviral IDS.ApoEII
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Conditions
mucopolysaccharidosis type 2
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