Papillon Therapeutics Receives Orphan Drug Designation from the U.S. Food and Drug Administration for PPL-001 for the Treatment of Friedreich's Ataxia

SAN DIEGO--(BUSINESS WIRE)--Sep 17, 2024-- Papillon Therapeutics Inc., today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Papillon’s PPL-001, an experimental treatment for Friedreich's ataxia. 

PPL-001 is an experimental gene-corrected CD34+ hematopoietic stem and progenitor cell (HSPC) therapy. This therapeutic’s novel approach utilizes targeted excision to correct the GAA repeat expansion in Intron 1 of the FXN gene.

 https://www.bakersfield.com/ap/news/papillon-therapeutics-receives-orphan-drug-designation-from-the-u-s-food-and-drug-administration-for/article_6df57ffb-45ae-5345-a0af-74d4d262b686.html

http://www.businesswire.com/news/home/20240917506319/en