mPPL-001

mPPL-001 is a murine analog of PPL-001, an autologous, ex vivo CRISPR/Cas9-edited hematopoietic stem and progenitor cell (HSPC) therapy developed for the treatment of Friedreich’s ataxia (FRDA). The therapy utilizes a high-fidelity CRISPR/Cas9 ribonucleoprotein complex containing two guide RNAs (UP4 and DN4) to target and excise pathogenic GAA repeat expansions in intron 1 of the frataxin (FXN) gene. By restoring FXN expression and reprogramming pathological inflammatory microenvironments, mPPL-001 has demonstrated the ability to rescue neurologic, cardiac, and pancreatic pathology in mouse models. The program is being advanced toward clinical evaluation, with PPL-001 representing the human therapeutic candidate that has successfully completed INTERACT and pre-IND meetings with the FDA.