Charles River Laboratories International, Inc. and INADcure Foundation, a nonprofit organization developing treatments for Infantile Neuroaxonal Dystrophy (INAD) and other forms of PLA2G6-related neurodegeneration (PLAN), entered into a plasmid DNA manufacturing alliance.

The collaboration will leverage Charles River’s contract development and manufacturing organization services in High Quality (HQ) plasmid DNA production, to manufacture its leading candidate for Phase I/II clinical trials. In INAD patients, a mutation or genetic alteration in the PLA2G6 gene causes mitochondrial and axonal membrane defects that lead to neuronal damage with axonal swellings and accumulation of pre-synaptic spheroids.

Like other neurodegenerative diseases, INAD is progressive, and symptoms include poor motor function, involuntary eye movements, seizures, hearing loss, and difficulty swallowing and breathing. INAD affects over 150 children worldwide with a life expectancy of 10 or less years.

Charles River’s plasmid DNA production, specifically HQ plasmid, combines key features of Good Manufacturing Practice (GMP) manufacture with rapid turnaround times to accelerate the timeline to clinic. Plasmid DNA is a critical starting material for many cell and gene therapy therapeutics and demand continues to outstrip supply.

In response to this, Charles River recently announced the opening of a state-of-the-art HQ plasmid manufacturing center of excellence to address these supply shortages and support the growing needs of the cell and gene therapy field.

In addition, Charles River, with the acquisitions of Cobra Biologics, Vigene Biosciences, and Cognate BioServices, in 2021, has extended its comprehensive cell and gene therapy portfolio to include CDMO capabilities spanning plasmid DNA, viral vector, and cellular therapy production for clinical through to commercial supply.

“The opportunity to work with INADcure is exactly why we do the work we do. Their work is incredibly important to patients with this ultra-rare disorder,” said Kerstin Dolph, corporate senior vice president, Biologics Solutions, Charles River. “We are excited for the INADcure team to achieve this next stage of treatment development.”

“Our collaboration with Charles River brings us closer to accomplishing our goal of delivering breakthrough treatments for INAD,” Leena Panwala, founder & executive director at INADcure. “We are excited to begin HQ plasmid DNA manufacturing to further develop our treatment with their experienced team. Their invaluable expertise will undoubtedly contribute to the advancement of our gene therapy program.”