Rocket Pharmaceuticals has recently dosed the first patient in their trial of RP-A501, a treatment for Danon disease. According to BioSpace, this trial will test the safety, tolerability, and efficacy of the gene therapy.
About Danon Disease
Danon disease is a rare condition that is characterized by cardiomyopathy, intellectual disabilities, and the progressive weakening of the skeletal muscles. Affected individuals will either experience dilated or hypertrophic cardiomyopathy, but both have the potential to cause heart failure. Other symptoms include chest pain, heart palpitations, breathing problems, issues with vision, cardiac preexcitation, and gastrointestinal disease. All of these are caused by mutations in the LAMP2 gene, which affects lysosomal function. Medical professionals are unsure of how exactly this results in the symptoms. They do know that it is inherited in an X-linked dominant pattern. As of now, there are no cures or treatments specific to Danon disease. Treatment is symptomatic and often requires a team of specialists.
RP-A501 for Danon Disease
The Phase 1, open-label trial will ensure that RP-A501 is safe and well tolerated, and it will also begin to evaluate the efficacy. Researchers will also be looking at muscle transduction, clinical stabilization, and histologic correction. This is the higher dose cohort, as the lower dose cohort was successfully completed and approved by the FDA. Male patients over the age of 15 will be treated with 1.1×1014 genome copies/kilogram of the gene therapy administered through an IV.
As there are currently no treatments specific to Danon disease, this gene therapy is very promising and exciting. Both medical professionals and patients are eagerly awaiting the results of this phase and all further stages in the trial. Hopefully, this treatment will receive FDA approval one day and be able to better the lives of those with Danon disease.