Clinical Trial Update: Gene Therapy for Pyruvate Kinase Deficiency (PKD) [Phase 1]

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Summary

This document provides an update on a Phase 1 gene therapy trial for Pyruvate Kinase Deficiency, noting that the Phase 1 study has completed.

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Diseases / indications

• Pyruvate Kinase Deficiency

Drugs mentioned

• Rocket Pharmaceuticals investigational gene therapy for Pyruvate Kinase Deficiency

Snippet

Sponsor: Rocket Pharmaceuticals Inc. | Conditions: Pyruvate Kinase Deficiency | Phase 1 | Status: COMPLETED