DB17647InvestigationalGene Therapies
RP-A501 is a gene therapy product consisting of a recombinant adeno-associated virus serotype 9 (rAAV9) capsid containing the human LAMP2B transgene (AAV9:LAMP2B), which encodes lysosome-associated membrane protein 2 (LAMP-2). It... It is being investigated for Danon disease.
- Also known as
- AAV9.LAMP2B · Adeno-associated virus serotype 9 vector containing the human lysosome-associated membrane glycoprotein 2 isoform B transgene (AAV9.LAMP2B)
- Code names
- RP-A501
What you can answer from here — as of September 25, 2025
Which companies are running trials of RP-A501, in which indications and phases, and which of those programs are still active?
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