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UCL, Syncona create new gene therapy biopharma

December 11, 2015
Life sciences

UCL Business, the wholly-owned technology transfer company of UCL, has teamed up with investment group Syncona to create a new biopharmaceutical company called Freeline Therapeutics, which will focus on development and commercialisation of gene therapies for bleeding and other debilitating disorders.

With an investment from Syncona of £25 million in Series A financing and from its base at UCL Royal Free London Hospital, the company will work on gene therapies based on its next-generation adeno-associated virus (AAV) gene therapy platform developed by Amit Nathwani, Professor of Haematology at UCL, the new firm’s chief scientific officer.

AAV is a small virus which infects humans but is not currently known to cause disease, causing a very mild immune response. Properties including its ability to infect both dividing and dormant cells make it an attractive candidate for creating viral vectors for gene therapy.

Freeline’s AAV gene therapy platform builds on the successful haemophilia B Phase I/II trial in which all ten treated haemophilia B patients showed safe, sustained expression of blood clotting Factor IX from a single treatment.

“It is exciting to be building a company on this ground-breaking technology that has the potential to transform the lives of patients with a single administration,” said chairman Chris Hollowood, also a Partner with Syncona LLP.

By Selina McKee

Source: Pharma Times

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