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Charles River Laboratories and Cure AP-4 will collaborate on gene therapy manufacturing for AP-4 hereditary spastic paraplegia.
Charles River Laboratories and Cure AP-4, a non-profit foundation dedicated to raising funds and awareness about adapter-protein 4 hereditary spastic paraplegia (AP-4 HSP), announced on Sept. 6, 2022 a manufacturing collaboration.
Patients afflicted with any of the AP-4 HSP genetic disorders may present symptoms including, global developmental delay, microcephaly, seizures, brain malformation, and hypotonia. According to a company press release, out of the 249 currently confirmed global AP-4 HSP cases, most patients experience low mobility in some or all extremities as the disorder progresses and are severely intellectually challenged.
Under the collaboration, Charles River will provide high quality (HQ) plasmid DNA for Cure AP-4’s Phase I/II gene therapy trials against AP-4 HSP. DNA plasmids are a critical starting material for many cell and gene therapy therapeutics, and demand continues to exceed supply. To address these supply shortages and support the growing needs of the cell and gene therapy field, Charles River has opened its new HQ plasmid DNA Center located in Cheshire, United Kingdom.
Source: Charles River Laboratories