RP-A501
AssetAlso AAV9.LAMP2B
Delivers functional LAMP2B to restore lysosomal function and autophagy in Danon disease.
- Stage
- Phase 2
- Modality
- Gene therapy
- Target
- Autophagy
- 1
- Indication
- 0
- Trials
- 1
- Developer
Indications
1Danon disease
Phase 2, businesswire.com, 7 Oct 2026
In the full profile
- Targets and what it contains3
- Owner and partners1
- Evidence for its targets3
- Aging relevance2
Every asset’s record: target, stage, trials and the evidence behind it.