Multi-Systemic Genetic Medicines

Papillon Therapeutics is a clinical-stage biotechnology company advancing a pipeline of multi-systemic genetic medicines directed at the underlying causes of inherited disease.

We focus on neurodegenerative disorders with a technology platform that enables durable expression of functional protein throughout the body, including the brain and central nervous system.

Gene Modified Stem Cells

Papillon has developed a technology platform for gene modified hematopoietic stem and progenitor cell (HSPC) therapies, leveraging established clinical procedures and a mature manufacturing industry.

Gene-modified HSPC differentiate within the body into macrophage and microglia, which engraft and deliver functional protein to damaged tissues such as the brain, spine, heart, muscle, pancreas, and others.

News

2024, OCT 21 - Papillon Therapeutics Receives Rare Pediatric Disease Designation from the U.S. Food and Drug Administration for PPL-002 for the Treatment of Danon Disease

2024, OCT 15 - Papillon Therapeutics Receives Rare Pediatric Disease Designation from the U.S. Food and Drug Administration for PPL-001 for the Treatment of Friedreich's Ataxia

2024, OCT 1 - Papillon Therapeutics Receives Orphan Drug Designation from the U.S. Food and Drug Administration for PPL-002 for the Treatment of Danon Disease

2024, SEP 17 - Papillon Therapeutics Receives Orphan Drug Designation from the U.S. Food and Drug Administration for PPL-001 for the Treatment of Friedreich's Ataxia

2024, APR 15 - NuFund Venture Group Invests in Papillon Therapeutics to Advance Multi-Systemic Genetic Medicines

2023, NOV 20 - UC San Diego Researchers Receive Close to $10M from California Stem Cell Research Agency

2023, AUG 29 - UC San Diego Launches Gene Therapy Initiative, Targeting Treatments for Rare Diseases

2023, AUG 17 - Could stem cell transplants help treat Alzheimer’s disease?

2023, AUG 09 - Stem Cell Therapy Rescues Symptoms of Alzheimer’s Disease

2023, MAY 18 - Positive Data from Phase 1/2 Clinical Trial of Investigational Gene Therapy for Cystinosis

2022, DEC 01 - Scientists Receive $4.8M to Pursue Gene Therapy for ‘Incurable’ Disease

2019, JUN 20 - CIRM Board Approves New Clinical Trial for Rare Childhood Disease

2016, SEP 21 - Rare, Life-Threatening Childhood Disease is the Focus of CIRM’s Most Recent Investment