Development and Translation of PPL-001, an autologous, gene-edited CD34+ hematopoietic stem cell Therapy for Friedreich’s Ataxia Anusha Sivakumar, Colin Exline, Stephanie Cherqui.
Here, we present a GMP-compliant, commercially/clinically scalable manufacturing process and safety data of PPL-001, aimed at initiating the first-in-human HSPC and genome editing clinical trial for FRDA.
These promising results of scale-up feasibility and safety support our efforts in
clinical translation of CRISPR/Cas9 gene edited CD34+ HSPCs as a novel treatment approach
for Friedreich’s ataxia.
