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Charles River and INADcure Announce Gene Therapy Manufacturing Collaboration

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Charles River Laboratories International, Inc. 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.

 

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