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

1

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.

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Assets