- Cellares and Papillon Therapeutics have entered a collaboration to automate the manufacturing of PPL-001, an investigational gene-corrected hematopoietic stem and progenitor cell (HSPC) therapy for Friedreich’s Ataxia.
- Cellares will transfer the manufacturing process to its Cell Shuttle® platform and support release testing using its Cell Q™ automated quality control system to support clinical development.
Cellares and Papillon Therapeutics have announced a collaboration to automate the manufacturing of PPL-001, Papillon’s investigational gene-corrected hematopoietic stem and progenitor cell (HSPC) therapy for Friedreich’s Ataxia. Under the agreement, Cellares will transfer the manufacturing process to its Cell Shuttle® end-to-end cell therapy manufacturing platform and provide release testing through its Cell Q™ automated quality control system. The collaboration supports the advancement of PPL-001 toward clinical development while highlighting Cellares’ contract manufacturing capabilities for cell therapies.
PPL-001 is designed to correct the GAA repeat expansion in Intron 1 of the FXN gene, the mutation responsible for more than 95% of Friedreich’s Ataxia cases. According to the companies, the investigational therapy has received both Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. Food and Drug Administration. Its development is supported in part by grants from the California Institute for Regenerative Medicine, the Friedreich’s Ataxia Research Alliance, and the National Institutes of Health.
The Cell Shuttle platform provides a closed-system, automated workflow intended to improve reproducibility, reduce process variability, and lower manufacturing costs for HSPC-based therapies. Cellares said the collaboration demonstrates its ability to support programs ranging from rare pediatric diseases to larger cell therapy applications through scalable manufacturing infrastructure. As an IDMO, the company provides integrated development and manufacturing services comparable to those offered by a CDMO for advanced therapy developers.
“Delivering consistent gene correction across a patient-derived CD34+ population requires a level of process control that manual manufacturing simply cannot guarantee. Cellares’ manufacturing platform enables that capability at scale and this collaboration is a critical step toward clinical development and future commercial launch.”
Carter Cliff, Chief Executive Officer of Papillon Therapeutics
