Charles River and INADcure Announce Gene Therapy Manufacturing Collaboration

Charles River Laboratories International and the INADcure Foundation, a nonprofit organization whose mission is to support the development of treatments for Infantile Neuroaxonal Dystrophy (INAD) and other forms of PLA2G6-related neurodegeneration (PLAN), today announced a plasmid DNA manufacturing alliance.

The collaboration will leverage Charles River’s market leading contract development and manufacturing organization (CDMO) expertise 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.

Leena and Anil Panwala founded the INADcure Foundation in 2016, following the diagnosis of their two-year-old daughter, Ariya. Upon discovering there was no dedicated organization or centralized effort driving INAD research forward, they launched the Foundation with the mission to accelerate research and drive drug discovery. After seven years of unwavering dedication and with the tremendous support of INAD-afflicted families worldwide, the Foundation is furthering the development of a gene therapy treatment specifically designed for children affected by this rare neurodegenerative disease.

INAD, otherwise known as phospholipase associated neurodegeneration (PLAN), is a rare disease with no known treatment or cure. 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.

“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. We are excited for the INADcure team to achieve this next stage of treatment development,” said Kerstin Dolph, Corporate Senior Vice President, Biologics Solutions, Charles River.

“Our collaboration with Charles River brings us closer to accomplishing our goal of delivering breakthrough treatments for INAD. 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,” said Leena Panwala, Founder & Executive Director at INADcure.