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114Aging Biotech
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RP-A501 (AAV9.LAMP2B)

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Pivotal Phase 2 study of systemic LAMP2B gene replacement in males with Danon disease.

NCT ID
NCT06092034
Sponsor
Rocket Pharmaceuticals Inc.
Phase
Phase 2
Indication
Danon disease; LAMP2B deficiency and autophagic-lysosomal dysfunction
Started
2023-09-05
Status
Recruiting