Cellares and Papillon Therapeutics have announced a strategic collaboration to automate the manufacturing of PPL-001, an investigational gene-edited cell therapy being developed to treat Friedreich’s ataxia (FA), a rare inherited neurodegenerative disease that currently has no approved curative treatment.
The partnership aims to improve the production of the experimental therapy by using Cellares’ automated manufacturing technologies, helping prepare the program for clinical development while laying the groundwork for future commercial production.
PPL-001 is a gene-corrected hematopoietic stem and progenitor cell (HSPC) therapy designed to address the underlying genetic cause of Friedreich’s ataxia rather than simply managing symptoms. The treatment uses targeted gene editing to correct a mutation in the FXN gene, specifically the GAA repeat expansion in intron 1, which is responsible for more than 95% of Friedreich’s ataxia cases.
Friedreich’s ataxia is a rare genetic disorder that affects multiple organ systems, including the brain, spinal cord, heart, skeletal muscles, and pancreas. Symptoms typically begin during childhood or adolescence and progressively worsen over time, leading to impaired coordination, difficulty walking, muscle weakness, heart disease, and other serious complications. Although treatments can help manage some symptoms, no therapy currently exists that corrects the genetic defect responsible for the disease.
Papillon’s investigational therapy has already received both Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA). Development of the program is also supported by grants from the California Institute for Regenerative Medicine (CIRM), the Friedreich’s Ataxia Research Alliance (FARA), and the National Institutes of Health (NIH).
Under the collaboration, Cellares will transfer Papillon’s manufacturing process to its Cell Shuttle® platform, an automated, end-to-end manufacturing system designed for cell therapies. The company will also use its Cell Q™ automated quality control platform to support product testing before release.
According to Cellares, replacing traditional manual manufacturing with an automated closed-system process is expected to improve consistency, reduce production variability, and lower manufacturing costs. These advantages are particularly important for complex gene-edited therapies, where precise handling of patient-derived cells is essential to ensure product quality and reproducibility.
Carter Cliff, Chief Executive Officer of Papillon Therapeutics, said the collaboration addresses one of the biggest challenges in bringing advanced gene therapies to patients.
“Friedreich’s ataxia is a devastating disease that most often begins in childhood and adolescence, and PPL-001 represents an innovative approach to treating its underlying genetic cause,” Cliff said. “Delivering consistent gene correction across a patient-derived CD34-positive cell population requires a level of manufacturing control that manual processes simply cannot guarantee. Cellares’ platform provides that capability at scale and represents an important step toward clinical development and future commercialization.”
For Cellares, the collaboration demonstrates the flexibility of its automated manufacturing technology across both rare disease programs and larger commercial indications.
Fabian Gerlinghaus, Co-founder and Chief Executive Officer of Cellares, said manufacturing challenges are often particularly difficult for developers of therapies targeting rare diseases.
“The cell therapy field often focuses on diseases with the largest patient populations, but rare disease developers face equally significant manufacturing hurdles,” Gerlinghaus said. “PPL-001 is a scientifically compelling program targeting a disease with no approved curative treatment. Our Cell Shuttle and Cell Q platforms are designed to provide the precision, consistency, and scalability needed to help therapies like this reach patients more efficiently.”
Cellares describes itself as the first Integrated Development and Manufacturing Organization (IDMO), offering automated manufacturing infrastructure for cell therapies from early clinical development through commercial production.
The collaboration also reflects growing industry efforts to automate cell therapy manufacturing, a process traditionally reliant on labor-intensive manual procedures that can increase production costs and limit scalability.
As Papillon advances PPL-001 toward clinical testing, automated manufacturing is expected to play an increasingly important role in ensuring consistent product quality while supporting future regulatory and commercial requirements.
If successful, PPL-001 could become one of the first gene-editing therapies designed to directly correct the genetic mutation responsible for Friedreich’s ataxia, offering new hope to patients affected by this progressive and life-limiting disorder.