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Papillon Therapeutics

San Diego, California, United Statespapillon-tx.com ↗

Ownership: Private
Development stage: Phase 1

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

Drug pipeline

4 assets

PPL-002

Preclinical

3 more assets in the full pipeline.

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