Syncona Founds Purespring Therapeutics to Develop Gene Therapies for Kidney Diseases

Rare Daily Staff

U.K-based Syncona has founded Purespring Therapeutics with approximately $60 million (£45.0 million) in a series A financing to develop novel AAV gene therapies for chronic kidney diseases.

Founded around the seminal work of Moin Saleem, professor of Pediatric Renal Medicine at the University of Bristol, Purespring will seek to advance gene therapies for the treatment of chronic renal diseases that are currently poorly addressed with existing treatments.

Purespring will also establish an in vivo functional screening platform, FunSel, to initially screen for protective factors that could have applications across several kidney diseases. FunSel was developed by Mauro Giacca, professor at King’s College London and the University of Trieste and a leader in gene therapy of cardiovascular disorders. FunSel will give Purespring a target discovery platform uniquely suited to systematically move gene therapy beyond monogenic disorders.

“Gene therapy has come of age in certain areas, but a major challenge in complex solid organs is to precisely target the genetic material to the correct cell type. Using accumulated expertise in the Bristol Renal research group we have solved this crucial hurdle, putting us in a position to deliver curative gene therapy to patients with chronic and intractable kidney diseases,” said Saleem. “Syncona have had the foresight to see this potential and partnering with their world-leading gene therapy experience is the best possible springboard to successfully bring this technology to patients.”

Syncona says its $60 million commitment will fund the company build-out and enable progression to clinical stage and will work closely with Purespring as it builds out its operations and management team. Syncona Chief Investment Officer Chris Hollowood, has been appointed as Chairman and Syncona Partner Dominic Schmidt will join the Board of Directors.

Syncona’s holding value in Purespring is $5.2 million (£3.9 million) following the investment of the first tranche of the series A commitment and, at the point all current commitments are invested, Syncona will have an 84 per cent stake in the business.

Purespring is the sixth gene therapy company founded by Syncona. They include Nightstar (inherited retinal diseases and sold to Biogen for $877.0 million, Freeline (chronic systemic disease), Gyroscope (retinal inflammation and one of the first companies globally to move gene therapy from rare disease to highly prevalent severe diseases with no treatment options), Orbit Biomedical Device (a leading sub-retinal surgical delivery platform for delivering gene therapies to the eye, since merged with Gyroscope) and SwanBio (a CNS gene therapy company).

Photo: Moin Saleem, professor of Pediatric Renal Medicine at the University of Bristol