Papillon Therapeutics, Inc.
Wednesday, June 05, 2024
Company Presentation
Cell and Gene Therapy and Genome Editing
Company Presentation Theater 1
Papillon Therapeutics is advancing genetic medicines for life threatening conditions with neurologic disease, 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. 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.
Company Website:
http://www.papillon-tx.com
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. PPL-001development is supported by ~$4.8 million in non-dilutive funding from the California Institute of Regenerative Medicine (CIRM) and ~$3 million from NIH.
Number Of Unlicensed Products (For Which You Are Seeking Partners):
PPL-001 targeting Friedreich's ataxia
PPL-002 targeting Danon disease
PPL-003 targeting Alzheimer's disease
Company HQ City
San Diego
Company HQ State
CA
Company HQ Country
United States
CEO/Top Company Official
Carter Cliff
Development Phase of Primary Product
Pre-Clinical
Primary Speaker
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