Charles River and Purespring collaborate on gene therapy manufacturing

By Jane Byrne

- Last updated on GMT

© GettyImages/magicmine
© GettyImages/magicmine

Related tags plasmid DNA Gene therapy

The plasmid CDMO alliance is aimed at supporting the first gene therapy platform targeting kidney diseases.

Purespring Therapeutics 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. It is the first company to treat kidney diseases by directly targeting the podocyte, a specialized kidney cell implicated in certain kidney diseases, through adeno associated virus (AAV) gene therapy.

The 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, maintains the UK pioneer.

Plasmid platform

The program is leveraging Charles River’s established plasmid platform, eXpDNA, as well as its plasmid DNA manufacturing experience.

Releasing details about eXpDNA last month, the organization claimed that it can significantly reduce plasmid development and production timelines while streamlining the development journey for cell and gene therapy and vaccine developers with a focus on product quality and consistency.

Continuing in that vein, the company said the platform leverages Charles River’s expertise in developing, manufacturing, and releasing more than 200 High Quality (HQ) and Good Manufacturing Practice- (GMP) compliant plasmid DNA batches. “eXpDNA supports a client’s plasmid DNA strategy by offering a proven, and standardized plasmid platform approach, suitable for plasmid DNA programs across various applications. The platform consists of an efficient and robust a plug-n-play screening toolbox for tackling challenging plasmids, phase-appropriate production with fit-for-purpose facilities, on-hand materials, and in-house analytics. The standardized process approach expedites batch turnaround times to five weeks for HQ plasmid and 10 weeks for GMP plasmid.”

Kerstin Dolph, corporate senior vice president, biologics solutions, Charles River, commenting on the tie-up with Purespring said that company’s targeted approach has the potential to radically change the treatment of kidney diseases.

“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,” commented Julian Hanak, CEO, Purespring, who was formerly head of global gene therapy development at Biogen.

Purespring was founded on the work of Moin Saleem, professor of pediatric renal medicine at the University of Bristol. An initial £45m ($US54.5m) commitment to Purespring from Syncona Ltd is enabling the gene therapy innovator to progress its assets to the clinic.

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