Charles River to Provide pDNA Manufacturing Services in Two Deals

Editor's note: this story was originally published on BioPharmInternational.com.

In back-to-back agreements, Charles River Laboratories International will provide plasmid DNA (pDNA) manufacturing services to Axovia Therapeutics and Ship of Theseus. Announced on April 9, 2024, the Axovia agreement involves the manufacture high quality (HQ) gene-of-interest plasmid, which will support Axovia’s development of gene therapies for ciliopathies, including Bardet-Biedl Syndrome (BBS). Under the Ship of Theseus agreement, announced on April 4, 2024, Charles River will offer its expertise to manufacture pDNA to serve as the active drug substance for that company's lead candidate.

Axovia is working on a lead program, AXV101, an adeno-associated virus serotype 9 (AAV9)-based gene therapy. The therapy targets retinal dystrophy associated with BBS. In preclinical studies, the gene therapy was shown to modify the underlying disease and halt retinal degeneration, stopping BBS-induced weight gain and the development of obesity. FDA granted orphan drug and rare pediatric disease designations to Axovia for AXV101. In the deal, Axovia will utilize Charles River’s plasmid platform, eXpDNA, and pDNA manufacturing expertise.

“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,” said Kerstin Dolph, corporate senior vice-president, Global Manufacturing, Charles River, in a company press release.

Ciliopathies are caused by cilia dysfunction resulting from genetic mutations that cause defective or dysfunctional cilia in many organs. Dysfunctional cilia can lead to blindness, deafness, chronic respiratory infections, kidney disease, heart disease, infertility, obesity, and diabetes. According to the press release, more than 20 ciliopathies have been identified. Collectively, they affect an estimated 1 in 1000 people.

“This collaboration brings us closer to accomplishing our goal to develop potentially transformative therapies for ciliopathies. We are excited to leverage Charles River’s expertise in plasmid DNA production to help drive our work forward,” said Victor Hernandez, co-founder and chief scientific officer, Axovia, in the release.

Meanwhile, Ship of Theseus, a therapeutics company specializing in degradation-resistant homeobox (HOX) family biologics, is developing a proprietary suite of biologics based on HOX family transcription factors. These transcription factors are responsible for directing the differentiation of tissue stem cells towards their respective lineages for the repair and maintenance of correct tissue and organ function. According to a company press release, “proprietary modifications may enable these regulators to be used as therapeutics.”

Under the collaboration, Charles River and Ship of Theseus will develop therapeutics for a broad variety of target indications such as diabetic and chronic wounds, neutropenia, psoriasis, androgenetic alopecia, women’s health and infertility, and epithelial cancers. This collaboration will be led by Charles River’s good manufacturing practice (GMP) pDNA contract development and manufacturing organization center of excellence in Keele, United Kingdom. The Keele site will provide pDNA backbone generation, plasmid synthesis, GMP master cell bank generation, pre-production evaluation, and GMP pDNA manufacture, including in-house release testing.

“Ship of Theseus is proud to collaborate with Charles River as our manufacturing partner of choice. We trust the team’s decades of success developing, producing, and reliably delivering plasmid DNA and look forward to bringing our therapies to patients,” said Jeremy Elser, founding CEO, Ship of Theseus, in the press release.

Source: Charles River Laboratories International for Axovia Therapeutics Deal and Ship of Theseus Agreement