Company intelligence / Profile preview
Papillon Therapeutics
San Diego, California, United Statespapillon-tx.com ↗
- Ownership
- Private
- Development stage
- Phase 1
01
Overview
Papillon's clinical stage program for Cystinosis reported positive data in a Phase 1/2 clinical trial. The company has received Orphan Drug Designation for PPL-001 (Friedreich's ataxia) and PPL-002 (Danon disease), as well as Rare Pediatric Disease Designation for both programs.
- Therapeutic areas
- Rare DiseasesNeurology
- Modalities
- Gene modified hematopoietic stem and progenitor cell (HSPC) therapyGenetic medicines
- Industry
- Biotech
02
Drug pipeline
4 assets3 more assets in the full pipeline.
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