Plasmid CDMO alliance supports first gene therapy platform targeting kidney diseases

Charles River Laboratories International, Inc. (NYSE: CRL) and Purespring Therapeutics, a pioneering gene therapy company focused on transforming the treatment of kidney diseases, today announced a plasmid DNA contract development and manufacturing organization (CDMO) collaboration.

Supporting the first gene therapy platform targeting renal diseases, which affect approximately 840 million people, or around 10 percent of the global population, the program leverages Charles River’s established plasmid platform, eXpDNA™, and decades of experience at the Company’s plasmid DNA manufacturing center of excellence.

Purespring is engaged in the development of novel therapies which have the potential to stop or significantly slow down chronic kidney diseases for which there is no current therapy available, except for dialysis or transplantation. With an innovative focus on the podocyte, a specialized kidney cell type implicated in many kidney diseases, Purespring’s Adeno Associated Virus (AAV) based gene therapy presents a lower-dose, local delivery approach which maximizes both safety and efficacy, as well as lowering the cost of goods.

Plasmid DNA CDMO Services

Expanding its comprehensive cell and gene therapy portfolio to span plasmid DNA, viral vector, and cell therapy production, through the acquisitions of Cobra Biologics, Vigene Biosciences, and Cognate BioServices in 2021, and in addition to recent expansion projects, Charles River offers end-to-end support and supply chain simplification for developers seeking to accelerate their program while ensuring the highest quality control.

The eXpDNA plasmid manufacturing platform builds on Charles River’s proven plasmid DNA CDMO capabilities and processes, fine-tuned over decades successfully supporting vaccine and advanced therapy clients through clinical trials and beyond.

For more information, watch Sustainable Plasmid DNA Strategies, a case study webinar presented by Andrew Frazer, PhD, Associate Director Scientific Solutions, Charles River, and Amanda Weiss, Vice President, Chemistry, Manufacturing and Controls (CMC), Purespring Therapeutics, available on demand.

Approved Quotes

• “Purespring’s unique and targeted approach has the potential to radically change the treatment of kidney diseases. Their work is incredibly important to patients worldwide, and we are thrilled to collaborate.” – Kerstin Dolph, Corporate Senior Vice President, Biologics Solutions, Charles River
• “Our team is proud to be leading a revolution in the treatment of kidney diseases and understands that a key factor to our success is developing a robust and reliable CMC platform. Purespring’s established relationship with Charles River leverages a breadth of contract development and manufacturing experience and expertise.” - Julian Hanak, Chief Executive Officer, Purespring Therapeutics

About Charles River

Charles River provides essential products and services to help pharmaceutical and biotechnology companies, government agencies and leading academic institutions around the globe accelerate their research and drug development efforts. Our dedicated employees are focused on providing clients with exactly what they need to improve and expedite the discovery, early-stage development and safe manufacture of new therapies for the patients who need them. To learn more about our unique portfolio and breadth of services, visit www.criver.com.

About Purespring Therapeutics

Purespring is the first company to treat kidney diseases by directly targeting the podocyte, a specialized kidney cell implicated in many kidney diseases, through AAV gene therapy.

Headed by former Biogen Head of Global Gene Therapy Development, Julian Hanak, Purespring was founded on the work of Professor Moin Saleem, Professor of Pediatric Renal Medicine at the University of Bristol, where he heads a world leading group researching glomerular diseases. Purespring seeks to advance gene therapies for the treatment of both monogenic and non-monogenic chronic renal diseases that are currently poorly addressed with existing treatments.

The company also has a proprietary in-vivo pipeline engine, FunSel, which is a library of 1,200 biological factors that could be candidates for gene therapy, combined with a screening method to evaluate these factors in disease models. FunSel allows Purespring to discover new gene therapy candidates across all indications, unconstrained by genetics, to find the right candidate to make the best therapy.

An initial £45 million commitment to Purespring from Syncona Ltd is enabling Purespring to progress its assets to the clinic. Syncona’s Chief Investment Officer, Chris Hollowood, serves as Chairman. For more information please visit: purespringtx.com and follow us on LinkedIn.

View source version on businesswire.com: https://www.businesswire.com/news/home/20230215005179/en/