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Phase I/II open-label dose-escalation study of RGX-111 intracisternal gene therapy in mucopolysaccharidosis type I
MOH_2022-04-28_010676Phase 2Completed
- Sponsor
- REGENXBIO Inc.
- Start
- Mar 2022
- Modality
- Recombinant Proteins and Enzymes, Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of RGX-111 in mucopolysaccharidosis type I, sponsored by REGENXBIO Inc. Started Mar 2022; status: completed.
02
Drugs
RGX-111
03
Conditions
mucopolysaccharidosis type I
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