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news.Cellares and Papillon Therapeutics have entered a partnership to automate the manufacturing of PPL-001, an investigational therapy developed by the latter for Friedreich’s ataxia (FA), a rare inherited neurodegenerative disorder.
Friedreich’s ataxia affects multiple organ systems, including the central nervous system, heart. Credit: evan_huang / Shutterstock.com.
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PPL-001 is a gene-corrected haematopoietic stem and progenitor cell (HSPC) therapy.
Under the collaboration, Cellares will adapt the PPL-001 manufacturing process for its Cell Shuttle platform, and support release testing through its Cell Q automated quality control system.
Both technologies are designed to increase consistency, reduce variability, and cut production costs of cell therapies that require precise handling.
FA affects multiple organ systems, including the central nervous system, heart, skeletal muscle, and pancreas.
The disorder commonly causes progressive loss of coordination and hypertrophic cardiomyopathy from childhood or adolescence.
PPL-001 targets the guanine-adenine-adenine (GAA) repeat expansion in the frataxin (FXN) gene, associated with more than 95% of FA cases, by using targeted gene editing in HSPCs.
It received orphan drug and rare paediatric disease designations from the US Food and Drug Administration (FDA).
The therapy’s development is also supported in part by grants from the California Institute for Regenerative Medicine, the FA Research Alliance, and the National Institutes of Health.
Papillon Therapeutics CEO Carter Cliff said: “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.
“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.”
Recently, Cellares and Sonoma Biotherapeutics entered a collaboration to automate the manufacturing of the latter’s lead cell therapy programme, SBT-77-7101, using the Cellares Cell Shuttle platform.
