Charles River Laboratories International, Inc. and Cure AP-4, a non-profit foundation dedicated to raising funds and awareness about Adapter-Protein 4 Hereditary Spastic Paraplegia (AP-4 HSP), have entered a manufacturing collaboration. Charles River, a contract research and development manufacturing organization (CRO/CDMO), will provide High Quality (HQ) plasmid DNA for Cure AP-4’s Phase I/II gene therapy trials against AP-4 HSP.
 
Cure AP-4’s gene therapy treatment will look to address the root cause of AP-4 HSP, a rare neurodegenerative disorder, and is intended as a one-time, curative treatment for the patient.
 

Plasmid DNA Manufacturing Services 

The collaboration will leverage Charles River’s expertise in plasmid DNA production, specifically HQ plasmid, which combines key features of GMP manufacture with rapid turnaround times to accelerate the timeline to clinic. DNA plasmids are a critical starting material for many cell and gene therapy therapeutics and demand continues to grow. Charles River recently announced the opening of a state-of-the-art HQ plasmid manufacturing center of excellence to address these supply shortages and support the growing needs of the cell and gene therapy field.
 
“The opportunity to work with a group as driven as Cure AP-4 is exactly why we do the work we do. Playing a small but critical role in delivering a potentially transformative therapy to a severely underserved patient population gives us an incredible sense of pride, and we are excited for the Cure AP-4 team to achieve this next stage,” said Birgit Girshick, Corporate Vice President and Chief Operating Officer, Charles River.
 
“We refuse to accept the bleak prognosis which our children face and have decided to fight. Our aim is to improve the quality of life for children impacted by AP-4 HSP by accelerating the research for cures and treatments and providing support for patient therapies critical to their well-being and rehabilitation. Our partnership with Charles River brings us closer to accomplishing that goal and we are excited to begin manufacturing our treatment with their experienced team,” said Chris Edwards, Robbie Edwards’ father and President, Cure AP-4.