Wellcome invests in gene therapy startup

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The Wellcome Trust has put up $38 million to launch a new company that will have a primary focus on developing a potential one-time treatment for hemophilia.

Freeline Therapeutics will be aided by Wellcome’s Syncona subsidiary and will have technology licensed from University College London, with hopes to build a clinical case for a treatment that could potentially reverse hemophilia B.

Hemophilia is brought on by the lack of key proteins that are needed for coagulation and Freeline’s top candidate uses a harmless strain of adeno-associated virus to correct this deficiency by delivering a healthy copy of the gene that will regulate a particular protein.

Developed by UCL Professor Amit Nathwani, the gene therapy reduced bleeding episodes by 90% during the course of 3 years in a ten-patient trial with no observed toxic effects.

“Freeline will bring industry-leading development people and our expertise together to ensure our clinical translation is rapidly converted to registered therapeutics”, Nathwani said in a statement.

Syncona partner Christian Groendahl will serve as as CEO of the company, with Nathwani joining as chief scientific officer. Freeline’s top priority is hemophilia, with further goals of using Nathwani’s viral platform for the treatment of other, unnamed diseases.

Source: Syncona and UCLB launch Freeline Therapeutics; Press Release