Charles River forms pDNA manufacturing partnerships with Axovia Therapeutics and Ship of Theseus
Charles River has secured two deals to provide plasmid DNA manufacturing services. One deal is with Axovia Therapeutics for their gene therapy under development, while the other is with Ship of Theseus for their lead candidate.
Charles River Laboratories International has established manufacturing agreements with Axovia Therapeutics and Ship of Theseus to provide plasmid DNA (pDNA) services to these companies. As part of the Axovia agreement, Charles River will be responsible for producing top-notch gene-of-interest plasmid. This will greatly assist Axovia in their efforts to develop gene therapies for ciliopathies, such as Bardet-Biedl Syndrome (BBS).
Charles River has entered into an agreement with Ship of Theseus, a therapeutics company focused on developing degradation-resistant homeobox (HOX) family biologics. As part of this collaboration, Charles River will utilize its expertise to manufacture pDNA, which will serve as the active drug substance for Ship of Theseus' leading candidate. The Ship of Theseus agreement was made public on April 4, 2024.
Axovia is currently developing a lead program called AXV101, which is a gene therapy based on adeno-associated virus serotype 9 (AAV9). The therapy focuses on addressing retinal dystrophy that is linked to BBS. Preclinical studies demonstrated that the gene therapy effectively altered the underlying disease, preventing retinal degeneration, and effectively stopping BBS-induced weight gain and the onset of obesity. Axovia has been granted orphan drug and rare pediatric disease designations by the FDA for AXV101. As part of the agreement, Axovia will be able to take advantage of Charles River's plasmid platform, known as eXpDNA, as well as their expertise in pDNA manufacturing.
“We are committed to supporting Axovia in the pursuit of creating treatment options for patients living with ciliopathies. The Charles River team brings decades of expertise in HQ plasmid development, and we look forward to using these capabilities to make a difference for patients,”stated Kerstin Dolph, corporate senior vice-president, Global Manufacturing, Charles River, in a recent company press release.
Ciliopathies arise from genetic mutations that impair cilia function, leading to defective or dysfunctional cilia in multiple organs. Malfunctioning cilia can result in a range of health issues, including vision and hearing impairment, recurring respiratory infections, kidney and heart problems, difficulties with fertility, as well as obesity and diabetes. As stated in the press release, over 20 ciliopathies have been discovered. Altogether, they impact approximately 1 in 1000 individuals.
Meanwhile, Ship of Theseus is working on creating a unique collection of biologics centered around HOX family transcription factors. These transcription factors play a crucial role in guiding the development of tissue stem cells into specific cell types, ensuring the proper functioning and repair of tissues and organs. As per a company press release, the regulators may have potential applications as therapeutics due to certain modifications.
Charles River and Ship of Theseus have joined forces to create therapeutics for a wide range of medical conditions, including diabetic and chronic wounds, neutropenia, psoriasis, androgenetic alopecia, women's health and infertility, and epithelial cancers. This collaboration will be overseen by Charles River's center of excellence for contract development and manufacturing of pDNA, located in Keele, United Kingdom. The Keele site offers a range of services including pDNA backbone generation, plasmid synthesis, GMP master cell bank generation, pre-production evaluation, and GMP pDNA manufacture, along with in-house release testing.
