PPL-002

PPL-002 is an experimental gene-modified CD34+ hematopoietic stem and progenitor cell (HSPC) therapy developed for the treatment of Danon disease. The therapy is designed to express functional Lamp-2 protein, which is critically deficient in individuals with Danon disease, a rare X-linked genetic disorder caused by mutations in the LAMP2 gene. By restoring Lamp-2 expression, PPL-002 aims to address the underlying cause of the disease and improve symptoms across multiple organ systems, including heart and skeletal muscle. Preclinical studies have shown that PPL-002 can improve disease phenotype in affected tissues and potentially modify or reverse progression of this multi-systemic disorder. The drug has received Orphan Drug Designation from the FDA and its development is partially funded by the California Institute for Regenerative Medicine (CIRM). Papillon Therapeutics leads its development.