Co-founder and Director
Papillon Therapeutics
Papillon Therapeutics develops gene-modified hematopoietic stem and progenitor cell (HSPC) therapies to deliver functional proteins to damaged tissues, including the brain and heart, addressing the underlying causes of neurodegenerative and systemic diseases. Their technology enables durable expression of therapeutic proteins throughout the body, targeting inherited conditions such as Friedreich's Ataxia and Danon Disease.
- Biotechnology
Updated 7 months ago
Funding
Funding rounds are not available yet.
Founders
Product
Problem
Many inherited diseases, including neurodegenerative disorders, lack effective treatments that address the underlying genetic causes and deliver therapeutic proteins durably to affected tissues, particularly the brain and central nervous system. Current approaches may not provide sufficient protein expression or distribution throughout the body to halt or reverse disease progression.
Solution
Papillon Therapeutics develops multi-systemic genetic medicines using gene-modified hematopoietic stem and progenitor cell (HSPC) therapies to treat inherited diseases. Their technology platform enables durable expression of functional proteins throughout the body, including the brain and central nervous system, by leveraging established clinical procedures and a mature manufacturing industry. Gene-modified HSPCs differentiate into macrophages and microglia, which engraft and deliver functional protein to damaged tissues such as the brain, spine, heart, muscle, and pancreas. This approach targets the underlying causes of inherited diseases, offering a potential for long-term therapeutic benefit.
Target Audience
The primary target audience includes patients with inherited diseases, particularly neurodegenerative disorders like Friedreich's Ataxia and Danon Disease, and the healthcare providers who treat them.
Features
- Gene-modified HSPC therapies for durable protein expression
- Targeted delivery of functional proteins to the brain, spine, heart, muscle, and pancreas
- Utilizes established clinical procedures and a mature manufacturing industry
- Focus on neurodegenerative disorders and inherited diseases
- Pipeline includes PPL-001 for Friedreich's Ataxia and PPL-002 for Danon Disease