The Latest Updates From the Gene-Editing Clinical...
Feb. 26, 2025
Relevant Interviews, Webinar and Trial Updates posted by CRISPR Medicine News.
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Papillon Therapeutics
Company Type: Therapeutic development
Main focus: Developing gene-edited haematopoietic stem and progenitor cell (HSPC) therapies to treat various disease
Company stage: Clinical
Diseases: Alzheimer's disease, cystinosis, Friedrich’s ataxia, Danon disease, and other genetic disorders
Genome-editing tool: CRISPR-Cas9
Funding stage: Private
Location: San Diego, California, USA
Website: www.papillon-tx.com
Pipeline: www.papillon-tx.com/pipeline
Partners:
Papillon Therapeutics is a biotechnology company focused on the development of gene-edited haematopoietic stem and progenitor cell (HSPC) therapies. The company’s approach involves using CRISPR-Cas9 technology to edit HSPCs to correct disease-causing mutations. Papillon’s lead pre-clinical programme, PPL-001, is a gene-edited HSPC candidate designed to correct the mutation located in the FXN gene of patients with Friedreich's ataxia.
