Charles River Laboratories International, Inc. and Genetic Cures for Kids Inc (GC4K), an Australian non-profit foundation focused on research programs and clinical trials to find cures for rare diseases, today announced a plasmid DNA contract development and manufacturing organization (CDMO) collaboration.
Founded in 2021 by Golden and Chris Whitrod, parents of now 4 year old Tallulah Moon, GC4K’s first mission, known as Our Moon’s Mission, is to develop a gene therapy for the treatment of Hereditary Spastic Paraplegia Type 56 (SPG56). SPG56 is a debilitating neurodegenerative disease that robs children of the ability to walk, talk, and move independently.
What is SPG56?
SPG56 manifests as a progressive neurological disease characterised by varying degrees of spasticity and muscle weakness. This degenerative disease typically begins with motor and cognitive regression in childhood and continuously worsens through life. Additional clinical manifestations include intellectual disability, dystonia, cerebellar ataxia, subclinical peripheral neuropathy, seizures, and visual impairment.
Tragically, there is currently no treatment for this ultra-rare genetic disease. In addition, limited access to genetic testing means diagnoses are hard to achieve and as such, fewer than one in 1 million people have ever been diagnosed with SPG56 globally. GC4K is leading a gene therapy program that aim not to only cure SPG56, but to create a replicable framework that paves the way to develop treatments for some of the 7,000 other genetic diseases in the world.
Plasmid DNA Manufacturing Services
GC4K will leverage Charles River’s established plasmid platform, eXpDNA™, and premier expertise in plasmid DNA production, including High Quality (HQ) plasmid which combines key features of good manufacturing practice (GMP) manufacture with a rapid turnaround to accelerate time to clinic. As a critical starting material in the development of cell and gene therapies, in 2022 Charles River opened a state-of-the-art HQ Plasmid Manufacturing Center of Excellence dedicated to addressing global supply shortages and support the growing needs of customers.
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