Disease Focus: Danon Disease
A Phase 2 Study Evaluating the Efficacy and Safety of IV Administered rAAV9 Vector Containing RP-A501 in Male Patients with Danon Disease
Genetically Modified Hematopoietic Stem Cells for the Treatment of Danon Disease
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 only existing treatment for Danon disease is cardiac transplant, this therapy would significantly meet an unmet need. It also may help many other similar diseases. […]
Ex Vivo Modified Hematopoietic Stem Cells to Treat Danon Disease
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 Action Engrafted HSPC progeny will supply normal LAMP2B to the heart, liver, muscle, and brain via lysosomal cross-correction. Specifically, macrophages transfer lysosomes containing LAMP2B to […]
A Phase 2 Study Evaluating the Efficacy and Safety of IV Administered rAAV9 Vector Containing RP-A501 in Male Patients with Danon Disease
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 consisting of a recombinant AAV9 vector engineered with genetic elements designed to ensure consistent delivery and expression of the LAMP2B transgene. The therapy is administered […]