Danon Disease - Healing Genes

Danon Disease

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Gene Therapy for Male Patients With Danon Disease Using RP-A501; AAV9.LAMP2B

Gene Therapy for Danon Disease (DD): A Clinical Study Evaluating the Infusion of a Recombinant Adeno-Associated Virus Serotype 9 (rAAV9) Capsid Containing the Human Lysosome-Associated Membrane Protein 2 Isoform B (LAMP2B) Transgene (RP-A501; AAV9.LAMP2B) in Male Patients With Danon Disease

Phase 1


The University of California San Diego in researching an investigational gene therapy to treat male patients with Danon Disease (DD). The treatment uses a genetically modified virus to deliver a gene for the human lysosome-associated membrane protein 2 isoform B (LAMP2B), which is the gene typically carrying the mutations which cause Danon disease. The researchers hope the virus will impart the gene to the patient after a single infusion, resulting in expression of the gene and heart muscle cell correction.

Follow up for 3 years will include lab testing and clinical assessments, including biopsies.


  • Be male and 8 years or older
  • DD diagnosis with LAMP2 mutations confirmed and clinical confirmation of cardiac involvement
  • Not have received cardiac transplant or other solid organ transplant
  • Be HIV-negative


  1. Screening before the treatment
  2. A single infusion of the gene therapy drug will be administered at one of two dose levels.
  3. Monitoring of the patient for a few hours after dosing, with lab assessments required for up to 3 years post treatment.


Trials will take place at the University of California, San Diego in San Diego, CA. Map.

Contact: Barry H Greenberg, MD  |  858-246-2987  |  [email protected]




Rocket Pharmaceuticals Inc.

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