Cellares, Papillon partner to automate manufacturing of gene-corrected HSPC therapy

Cellares and Papillon Therapeutics 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.

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.

Friedreich’s ataxia 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 and rare pediatric disease designations from the FDA.

Earlier this month, Cellares’ smart factory in Bridgewater, NJ was one of seven facilities chosen to participate in the new FDA PreCheck Pilot Program, an initiative designed to strengthen domestic drug manufacturing.