Charles River Laboratories International, Inc. and Genetic Cures for Kids Inc (GC4K) have jointly revealed a groundbreaking collaboration in plasmid DNA contract development and manufacturing organization (CDMO). GC4K, a non-profit foundation based in Australia, is committed to pioneering research programs and clinical trials to discover cures for rare diseases.
Established in 2021 by Golden and Chris Whitrod, the parents of 4-year-old Tallulah Moon, GC4K's inaugural mission, known as Our Moon’s Mission, aims to develop a gene therapy for treating Hereditary Spastic Paraplegia Type 56 (SPG56). SPG56 is a severe neurodegenerative disease that deprives children of their ability to walk, talk, and move independently.
SPG56 manifests as a progressive neurological ailment characterized by varying degrees of spasticity and muscle weakness. Commencing with motor and cognitive regression in childhood, this degenerative disease persists throughout life, presenting additional clinical challenges such as intellectual disability, dystonia, cerebellar ataxia, subclinical peripheral neuropathy, seizures, and visual impairment.
Regrettably, there is currently no treatment available for this ultra-rare genetic disease. Compounded by limited access to genetic testing, the difficulty of diagnoses is underscored by fewer than one in 1 million people globally ever receiving an SPG56 diagnosis. GC4K spearheads a gene therapy program not only to cure SPG56 but also to establish a reproducible framework, paving the way for treatments for some of the 7,000 other genetic diseases globally.
In delivering on this ambitious mission, GC4K will leverage Charles River’s well-established plasmid platform, eXpDNA™, and its superior expertise in plasmid DNA production. This includes the utilization of High-Quality (HQ) plasmid, combining essential features of good manufacturing practice (GMP) with a rapid turnaround to expedite the time to clinic. Recognizing the pivotal role of plasmid DNA in developing cell and gene therapies, Charles River inaugurated a state-of-the-art HQ Plasmid Manufacturing Center of Excellence in 2022. This facility is dedicated to addressing global supply shortages and supporting the escalating needs of customers in the realm of gene therapy.
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