Papillon Therapeutics, Inc.
Monday, February 26, 2024
Papillon Therapeutics is advancing gene therapy treatments for life threatening conditions with neurologic disease, including Friedreich's ataxia, Danon disease, and Alzheimer’s disease. We specialize in developing gene modified hematopoietic stem and progenitor cell (HSPC) therapies, leveraging the natural capacity of these unique human cells to safely deliver a gene therapy throughout the body. Our clinical stage program, an 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. Non-dilutive funding awarded by the California Institute of Regenerative Medicine (CIRM) to our scientific founders totals $28.7 million towards the Cystinosis, Friedreich's ataxia, and Danon disease programs.
CEO/Top Company Official
Carter Cliff CEO
Lead Product in Development
PPL-001 is a gene modified hematopoietic stem and progenitor cell (HSPC) designed to correct the mutation located in the FXN gene of patients with Friedreich's ataxia. Patients receiving gene modified HSPC treatments undergo a standard procedure of HSPC mobilization and collection, followed by gene modification, cell processing, and infusion. Our approach aims to address the underlying cause of the condition through correction of the GAA repeats in the FXN Intron 1, yielding an HSPC that delivers functional Frataxin throught the body.
Development Phase of Primary Product
Number Of Unlicensed Products