Cellares and Papillon Therapeutics Partner to Automate Manufacturing of PPL-001 for Patients With Friedreich’s Ataxia

Collaboration will apply Cellares’ Cell Shuttle® and Cell Q™ platforms to PPL-001, an investigational hematopoietic stem cell therapy for a rare multi-systemic pediatric disease

SOUTH SAN FRANCISCO, Calif. & SAN DIEGO--(BUSINESS WIRE)--Cellares, the first Integrated Development and Manufacturing Organization (IDMO), and Papillon Therapeutics Inc., a clinical-stage biotechnology company advancing a pipeline of multi-systemic genetic medicines directed at the underlying causes of inherited disease, today announced a collaboration to automate manufacturing of PPL-001, Papillon’s investigational gene-corrected hematopoietic stem and progenitor cell (HSPC) therapy targeting Friedreich’s ataxia.

Friedreich’s ataxia (FA) is a rare, inherited neurodegenerative disorder that affects multiple organ systems simultaneously, including the central nervous system, heart, skeletal muscle, and pancreas, and typically causes progressive loss of coordination and hypertrophic cardiomyopathy beginning most often in childhood and adolescence. PPL-001 uses targeted gene editing to correct the GAA repeat expansion in Intron 1 of the FXN gene, the mutation responsible for more than 95% of FA cases. The therapy has received both Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA, and its development is supported in part 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 translate the PPL-001 manufacturing process onto the Cell Shuttle®, its end-to-end cell therapy manufacturing platform, and support release testing through the Cell Q™, its automated quality control and release testing system. The Cell Shuttle’s closed-system, automated workflow is designed to improve reproducibility, reduce process variability, and lower manufacturing costs for HSPC-based therapies that require precise handling and analytical control at each stage of production.

“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 partnership reflects Cellares’ ability to support cell therapy programs across a broad spectrum of manufacturing scales, from rare pediatric indications with small patient populations to large autoimmune programs requiring hundreds of thousands of doses per year. For developers like Papillon, Cellares provides flexible manufacturing infrastructure and predictable economies of scale required to support both near-term clinical trials and global commercial production.

“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.”

About Papillon Therapeutics

Papillon is dedicated to delivering cures from within by harnessing the body’s own cells. Overcoming the targeting barriers of traditional genetic medicines, the company’s proprietary platform utilizes patient-derived hematopoietic stem cells to systemically distribute functional proteins across multiple affected organs. Papillon’s lead program, PPL-001, is a one-time, potentially lifetime-durable treatment designed to arrest the progression of Friedreich’s ataxia across affected tissues—including the brain, spine, heart, and pancreas. Validated by a landmark clinical partnership with Novartis, Papillon is advancing a pipeline of transformative cell therapies engineered to restore health across a lifetime. For more information, visit https://papillon-tx.com/ and follow Papillon on LinkedIn.

About Cellares

Cellares is the first Integrated Development and Manufacturing Organization (IDMO), providing global cell therapy development and manufacturing services through an Industry 4.0 approach to the mass manufacture of the living drugs of the 21st century. The company enables drug sponsors to develop, scale, and commercialize cell therapies with the capacity, reliability, and economics required to meet total patient demand.

Cellares' fully automated platforms — Cell Shuttle® for end-to-end cell therapy manufacturing and Cell Q™ for automated in-process and release quality control — are deployed across its network of IDMO Smart Factories worldwide. These technologies deliver industry-leading manufacturing economics, higher process success rates, and the ability to produce up to 10× more cell therapy batches than conventional CDMOs with comparable footprint and headcount, resulting in the lowest cost of manufacturing in the industry. The Cell Shuttle is the first cell therapy manufacturing platform to receive the FDA's Advanced Manufacturing Technology (AMT) designation, and has demonstrated a 100% automation success rate across more than a dozen automated processes.

Cellares has achieved key clinical validation milestones, including a successful IND Amendment enabling active clinical manufacturing on the Cell Shuttle platform, and the successful dosing of first patients in a partner clinical trial — marking the platform's transition from development-stage technology to clinically validated manufacturing infrastructure. These milestones span multiple therapeutic areas and cell therapy modalities, including both oncology and autoimmune indications.

Headquartered in South San Francisco, California, Cellares operates its first commercial-scale IDMO Smart Factory in Bridgewater, New Jersey, with additional facilities under construction in Europe and Japan. Through its global manufacturing network, Cellares is purpose-built to support both clinical and commercial programs and to expand access to life-saving cell therapies worldwide. For more information, visit www.cellares.com and follow Cellares on LinkedIn.