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OTL-101 gene therapy for ADA-SCID
2017-001275-23Phase 2Unknown
- Sponsor
- Great Ormond Street Hospital
- Start
- Jul 2017
- Modality
- Stem Cell Therapies → Cell Therapies, Gene Therapies
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of simoladagene autotemcel in adenosine deaminase deficiency severe combined immunodeficiency (ADA-SCID) and adenosine deaminase deficiency, sponsored by Great Ormond Street Hospital. Primary endpoint: Overall survival and event-free survival at 12 months. Status: unknown.
02
Drugs
simoladagene autotemcel
03
Conditions
adenosine deaminase deficiency severe combined immunodeficiency (ADA-SCID)adenosine deaminase deficiency
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