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Long-Term Follow-Up for Gene Therapy of Leukocyte Adhesion Deficiency-I
2022-501086-41-00Phase 2Authorised
- Sponsor
- Rocket Pharmaceuticals, Inc.
- Start
- Jul 2023
- Modality
- Stem Cell Therapies → Cell Therapies, Gene Therapies
- Countries
- 3
01
Overview
Phase 2, open-label, single-arm trial of marnetegene autotemcel in Leukocyte Adhesion Deficiency Type 1 (LAD1), sponsored by Rocket Pharmaceuticals, Inc. Primary endpoints: Significant infections requiring hospitalization or intravenous antimicrobials, New LAD-I-related skin or oral lesions and 13 more. Planned start Jul 2023; status: authorised.
02
Drugs
marnetegene autotemcel
03
Conditions
Leukocyte Adhesion Deficiency Type 1 (LAD1)
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