Partnership aims to automate production of ataxia cell therapy
Papillon teams with Cellares for FA stem cell therapy manufacturing
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A new partnership aims to provide fast, consistent production of an experimental cell therapy for Friedreich’s ataxia.
Papillon Therapeutics is teaming up with biological manufacturing company Cellares to automate the production of Papillon’s PPL-001, preparing the treatment for testing in clinical trials, and for commercial manufacturing if the treatment is ultimately approved for use, according to a press release from the companies.
Friedreich’s ataxia is caused by mutations that result in low levels of the protein frataxin, which is vital for the health and function of mitochondria (the so-called powerhouses of the cell that are key for cellular energy production). Lacking frataxin, mitochondria cannot generate energy efficiently, leading to problems in many types of cells, primarily nerves and muscle cells.
The vast majority of people with Friedreich’s ataxia carry a type of mutation called a trinucleotide repeat expansion. In this type of mutation, three nucleotides (the building blocks of DNA) are repeated an excessive number of times. This abnormal repeating disrupts the activity of the gene that encodes frataxin, ultimately leading to low levels of the protein.
‘Innovative approach’
“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, CEO of Papillon.
Stem cells are responsible for making new blood and immune cells in bone marrow. PPL-001 involves collecting these stem cells from patients, then using genetic editing to correct the disease-causing repeat expansion mutation. The modified cells are then returned to the patient.
The therapy’s goal is to allow the body to make blood and immune cells with functional frataxin protein. And since these cells travel through the bloodstream to reach all parts of the body, they can act as delivery vehicles to transport the working protein throughout the body. PPL-001 has shown promise in preclinical tests, and U.S. regulators have granted it rare pediatric disease and orphan drug designations, which aim to incentivize the development of treatments for rare diseases.
Cellares will provide manufacturing infrastructure to help ensure that the genetically engineered cells in PPL-001 are produced consistently and safely.
“Delivering consistent gene correction across a patient-derived [bone marrow stem cell] 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,” Cliff said.
The collaboration will use Cellares’ manufacturing program, Cell Shuttle, and its Cell Q quality control system.
“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 CEO 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 [stem and progenitor cell] therapy requires to reach more patients faster.”
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