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.

Mitotech Presents Promising Results in Friedreich's Ataxia

LONDON, Sept. 17, 2024 /PRNewswire-PRWeb/ -- Mitotech Ltd, a mitochondria-focused clinical-stage biotechnology company, presented new data for its mitochondria-targeted ferroptosis inhibitor in Friedreich's Ataxia (FA) model at the 149th annual meeting of the American Neurological Association. The conference took place on September 14-17, 2024, in Orlando, FL. 

Those new data demonstrated high level of efficacy of the company's lead compound SkQ1 – a mitochondria-targeted ferroptosis inhibitor – against ferroptosis and oxidative stress at strikingly low doses.The Friedreich's Ataxia-specific model has not only demonstrated SkQ1's great potential as a monotherapy for FA, but also highlighted its MoA as complementary to that of the currently available therapy – omaveloxolone. The latter aspect of our latest dataset opens the door for studying SkQ1 in a combination treatment in the clinic,"