Purespring is one of the first AAV gene therapy companies focused on the kidney. It has been founded around the seminal work of Professor Moin Saleem, Professor of Paediatric Renal Medicine at the University of Bristol, where he heads a world leading group researching glomerular diseases. Purespring will seek to advance gene therapies for the treatment of chronic renal diseases that are currently poorly addressed with existing treatments.
The company 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 has been developed by Professor Mauro Giacca, a leader in gene therapy of cardiovascular disorders, and provides Purespring with a target discovery platform uniquely suited to systematically move gene therapy beyond monogenic disorders.
Syncona’s £45.0 million commitment to Purespring will fund the company build-out and will enable it to progress to clinical stage. Syncona’s Chief Investment Officer, Chris Hollowood, has been appointed as Chairman and Syncona Partner Dominic Schmidt will be joining the Board of Directors. The Syncona team will work closely with Purespring as it builds out its operations and management team.
Dominic Schmidt, Partner, Syncona Investment Management Limited, said: “We believe that the foundation of Purespring represents a unique opportunity to build the global leader in renal gene therapy, where we will have a strong early mover advantage combined with a bespoke platform and world-class individuals. It is one of the first companies targeting the kidney using AAV gene therapy and will seek to treat a number of debilitating chronic diseases that are currently poorly addressed with existing therapies in line with Syncona’s ambitions to deliver transformational treatments to patients.”
Chris Hollowood, CIO, Syncona Investment Management Limited, said: “Purespring is the sixth gene therapy company to be founded by Syncona and clearly demonstrates our proprietary company creation approach. In Moin and his team, we are collaborating with clinical and scientific leaders and working in target tissue amenable to gene therapy, whilst the collaboration with Mauro provides a path for gene therapy to fulfil its promise in highly prevalent chronic degenerative conditions. We look forward to building a world class company around this innovative science, in order to develop therapies with the potential to deliver dramatic impact for patients. Purespring is an exciting addition to our gene therapy platform, where we are strategically positioned with significant expertise in building fully integrated platform companies.”
Professor Moin Saleem, Head of Bristol Renal, said: “This is an incredible opportunity to develop transformational treatments for kidney disease. 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. 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.”
Professor Mauro Giacca, Professor at King’s College London and the University of Trieste and former Director-General of ICGEB, said: “I am very excited to take part in this brilliant new initiative. Not only do AAV vectors hold tremendous promise for the genetic therapy of kidney hereditary disorders but they are also unique tools for the discovery of new therapeutics for common degenerative conditions.”
Syncona has significant expertise in gene therapy and Purespring is the sixth Syncona gene therapy company to be founded since 2012 underlining the Company’s leadership position in gene therapy. Each Syncona gene therapy company has had a distinct product focus and domain expert teams that focus on high quality and rapid execution.
Alongside Purespring, Syncona’s gene therapy companies have included: 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).