Wednesday, June 24th, 2026
11:00 AM - 12:00 PM ET
Friedreich's ataxia (FRDA) is a progressive neurodegenerative disorder caused by GAA repeat expansions in the frataxin (FXN) gene, leading to neurological dysfunction, muscle weakness, and cardiomyopathy. Despite advances in understanding disease biology, effective disease-modifying therapies remain limited. Recent developments in gene editing and stem cell engineering have created new opportunities to address the underlying genetic cause of FRDA through autologous cell-based therapeutic approaches.
In this webinar, brought to you by Innovative Research and Bio-Techne, Stephanie Cherqui, professor in the Department of Pediatrics at the University of California San Diego and director of the UCSD Gene Therapy Initiative, will discuss the development of PPL-001, a CRISPR/Cas9-edited CD34+ hematopoietic stem cell therapy for Friedreich's ataxia. The session will examine manufacturing development, scale-up strategies, safety evaluation, and preclinical efficacy studies supporting clinical translation. The discussion will also highlight approaches for assessing off-target editing, genomic stability, and regulatory readiness as part of efforts to advance this therapy toward first-in-human clinical evaluation.
Topics to be covered
- Principles of computer-assisted retrosynthesis and route planning
- Strategies for evaluating synthetic feasibility during early discovery
- Approaches for exploring alternative synthetic pathways
- Applications of retrosynthesis software in medicinal chemistry workflows
- Considerations for integrating digital synthesis planning into research environments
 | Stephanie Cherqui, PhD |
