Initial work with CGT Catapult has led to Purespring Therapeutics being awarded £45m from Syncona

Purespring Therapeutics, a University of Bristol spin-out company was able to achieve one of the largest single investments made to a new UK university biotech company.

The project

A University of Bristol spin-out company Purespring Therapeutics was seeking to advance and commercialise its ground-breaking gene therapy technology for chronic kidney diseases. The project aimed to address a global unmet need for renal conditions by directly targeting the glomerulus in the kidney, which could see treatment progress from lab to patients in three to four years. The company required investment to commercialise the research pioneered by Professor Moin Saleem, Professor of Paediatric Renal Medicine at Bristol Medical School. Professor Saleem’s work is the only study to date to have successfully demonstrated disease rescue in animal models, using this technique for a kidney disorder called nephrotic syndrome.

The University team felt they needed expert input on pre-clinical development plans to efficiently progress the opportunity towards clinic. Professor Saleem and Andrew Wilson, Senior Research Commercialisation Manager at the University of Bristol approached CGT Catapult as the obvious partner to support the project.


CGT Catapult’s involvement

The Commercialisation of Research team at CGT Catapult and the University of Bristol determined that a gap analysis on the Bristol team’s current plan and creation of a new pre-clinical development plan was required. CGT Catapult helped to prepare a submission to the MHRA Innovation Office and led the interaction, the outcome from which helped to validate the assumptions of the new development plan and provided the University of Bristol with a clear route to human clinical trials of their technology. CGT Catapult and the University of Bristol also worked together on costing the pre-clinical development plan which further supported interactions with Purespring Therapeutics’ investors. CGT Catapult’s support was provided over an eighteen-month period by experts with experience in ATMP development, the design of non-clinical safety studies, ATMP manufacturing, product QC and ATMP specific regulations.  The CGT Catapult team were able to connect the project to CDMOs in their UK network, continually opening doors and looking for enabling opportunities.

 

What this collaboration achieved

Through translating their research into a more investment-ready opportunity and gaining MHRA guidance on pre-clinical plans, this collaboration supported the interactions with investors which ultimately led to the creation of Purespring Therapeutics, who have recently secured a £45 million commitment from global healthcare company Syncona, in one of the largest single investments made to a new UK university biotech company.

Through this investment, Purespring Therapeutics will continue to develop its innovative gene therapies, which could see treatment progress from lab to patients in three to four years. The company will also have access to an in-vivo functional screening platform, FunSel, to screen for cell-specific protective factors delivered via gene therapy. Both Professor Saleem and Andrew Wilson credited the support and enthusiasm of the CGT Catapult team as a key factor behind enabling them to secure this investment, and in bridging the gap between their academic research and commercial opportunity.
 

Andrew Wilson,Senior Research Commercialisation Manager at the University of Bristol commented:

“We were looking for a consultant to provide us with a development plan for this research and the obvious place to go to was CGT Catapult. One of the things which really pleased me about this collaboration was their shared excitement around our ground-breaking technology. Importantly we were able to access a diverse range of expertise that probably isn’t available elsewhere. The CGT Catapult interaction has gone way beyond the input originally envisaged. The support which we have received from Jonathan Appleby and the team at CGT Catapult has been nothing short of fantastic. We are already working with CGT Catapult on other projects.”


This investment is reflective of the trust which Syncona has placed in cell and gene therapies, in addition to the increased attractiveness of cell and gene therapies from an investment standpoint. The project also represents the first-time gene therapies are being used to combat kidney diseases, and the work done by Purespring Therapeutics reflects a paradigm shift on how these diseases will be treated going forward.
 

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