Danon Disease

Genetically Modified Hematopoietic Stem Cells for the Treatment of Danon Disease

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Post published:April 4, 2025
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Research Objective We propose to discover a novel, genetically modified hematopoietic stem cell based treatment for Danon disease, a rare lysosomal storage disease that affects the heart. Impact As the…

Ex Vivo Modified Hematopoietic Stem Cells to Treat Danon Disease

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Post published:April 4, 2025
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Translational Candidate The candidate is CD34+ HSPCs transduced ex vivo with a LAMP2 lentiviral vector. Area of Impact Danon Disease, Lysosomal Storage Diseases, Drug Development for Rare Disease Mechanism of…

A Phase 2 Study Evaluating the Efficacy and Safety of IV Administered rAAV9 Vector Containing RP-A501 in Male Patients with Danon Disease

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Post published:April 4, 2025
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Therapeutic Candidate or Device RP-A501, a recombinant Adeno-Associated Virus Serotype 9 containing the LAMP2B transgene Indication Danon Disease Therapeutic Mechanism The proposed product, RP-A501, is an in vivo gene therapy…

A Phase 2 Study Evaluating the Efficacy and Safety of IV Administered rAAV9 Vector Containing RP-A501 in Male Patients with Danon Disease

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Post published:September 12, 2024
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