SOUTH SAN FRANCISCO, Calif., and SAN DIEGO — Cellares and Papillon Therapeutics have formed a partnership to automate manufacturing of PPL-001, an investigational gene-corrected stem cell therapy for Friedreich’s ataxia.
Under the collaboration, Cellares will transfer the PPL-001 manufacturing process to its Cell Shuttle platform and provide release testing through its automated Cell Q quality-control system.
PPL-001 is a hematopoietic stem and progenitor cell therapy designed to address the underlying genetic cause of Friedreich’s ataxia, a rare inherited neurodegenerative disorder that affects the nervous system, heart, skeletal muscles and pancreas. The disease often begins during childhood or adolescence and can cause progressive loss of coordination and hypertrophic cardiomyopathy.
The therapy uses targeted gene editing to correct a GAA repeat expansion in the FXN gene, which Papillon said is responsible for more than 95% of Friedreich’s ataxia cases.
PPL-001 has received Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. Food and Drug Administration. Its development is also supported by grants from the California Institute for Regenerative Medicine, the Friedreich’s Ataxia Research Alliance and the National Institutes of Health.
Cellares said its closed and automated Cell Shuttle workflow is designed to improve manufacturing consistency, reduce process variability and lower production costs. The platform will be used alongside Cell Q to support quality-control and product-release testing.
“Friedreich’s ataxia is a devastating disease that begins most often in childhood and adolescence, and PPL-001 represents an innovative approach to addressing its underlying genetic cause,” said Carter Cliff, Chief Executive Officer of Papillon Therapeutics. “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.”
The companies said the partnership is intended to support both clinical development and potential future commercial production of PPL-001.
“The cell therapy field tends to focus on the largest patient populations, but the manufacturing challenges facing rare disease developers are just as real and often harder to solve,” said Fabian Gerlinghaus, Co-founder and Chief Executive Officer of Cellares. “PPL-001 is a scientifically compelling program targeting a disease with no approved curative treatment, and the Cell Shuttle and Cell Q are built to deliver the precision and process consistency that a gene-corrected HSPC therapy requires to reach more patients faster.”
Papillon is developing PPL-001 as a one-time treatment intended to slow or stop disease progression across multiple affected tissues, including the brain, spinal cord, heart and pancreas.
