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SPK-3006 single-dose gene therapy study in adults with late-onset Pompe disease
NL-OMON55024Phase 2Not yet recruiting
- Sponsor
- Spark Therapeutics
- Start
- Apr 2021
- Modality
- Gene Therapies
- Countries
- 1
01
Overview
Phase 2, open-label trial of vanglusagene ensiparvovec in Pompe disease, sponsored by Spark Therapeutics. Primary endpoints: Safety and tolerability and Immune responses to vector and transgene. Planned start Apr 2021; status: not yet recruiting.
02
Drugs
vanglusagene ensiparvovec
03
Conditions
Pompe disease
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