We specialize in developing gene-modified hematopoietic stem and progenitor cell (HSPC) therapies, leveraging the natural capacity of these unique human cells to perform multi-systemic repair of damaged tissues.
Our pipeline includes a partnered Ph1/2 clinical stage program that recently reported positive data for a gene-modified HSPC therapeutic candidate, and multiple preclinical stage programs that benefit from this clinical program.
Papillon Therapeutics leverages gene-modified hematopoietic stem and progenitor cell (HSPC) platform technology to advance disease modifying treatments for life-threating inherited genetic disorders.