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RP-A501

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LAMP2B gene-replacement therapy in a resumed pivotal Phase 2 study for Danon disease.

Modality
systemic AAV9 gene therapy delivering the LAMP2B isoform
Availability (US)
Pivotal Phase 2 Danon-disease study Recruiting; enrollment and dosing continuing under modified protocol
Biomarkers
myocardial LAMP2 protein expression; left-ventricular mass index; cardiac biomarkers; safety and immune responses
Hallmark targeted
autophagy-lysosomal dysfunction, impaired proteostasis, cardiomyopathy
Kind
investigational gene therapy
Clinical trials
NCT06092034 — Phase 2, Recruiting on May 4, 2026 registry update