Papillon Therapeutics
Company Details
Status: Private
Employees: 1-10
Location:
3210 Merryfield Row, JLABS, Papillon Therapeutics, San Diego, California 92121, US
Type:
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Technology:
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About: Papillon Therapeutics is a clinical stage biotechnology company advancing multi-systemic genetic medicines for diseases with neurologic, cardiac, and other tissue pathologies, including Friedreich's ataxia, Danon disease, and Alzheimer's disease.
Inherited conditions often cause degeneration of multiple tissues throughout the body, inspring our approach to develop therapies that offer patients lifelong multi-systemic therapeutic benefit from a single treatment.
Our clinical stage program, a gene modified hematopoietic stem and progenitor cell (HSPC) therapy for treatment of Cystinosis, reported positive data in a Phase 1/2 clinical trial and is being advanced by Novartis.
Building on this success, we are advancing gene modified HSPC treatments for life threatening conditions with neurologic disease, including Friedreich's ataxia, Danon disease, and Alzheimer's disease.
Funding awarded by the California Institute of Regenerative Medicine (CIRM) and NIH to our scientific founders totals over $30 million towards the Cystinosis, Friedreich's ataxia, and Danon disease programs.
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Papillon Therapeutics | Active clinical trials on ClinicalTrials.gov
Active Clinical Trials Count | 0
No Active Trials Found On ClinicalTrials.gov.