The UK gene therapy biotech Freeline Therapeutics is to receive an investment worth €72M (£61.1M) from the UK VC firm Syncona in a Series C round to fund the clinical development of gene therapies for the blood disorder hemophilia B, Fabry disease, and other rare conditions.

Of the €72M commitment, Syncona has already wired €36M to Freeline. Freeline will receive the rest of the money at an undisclosed later date.

The first transfer will help Freeline to advance the clinical development of its lead gene therapy, which is in a phase I/II trial for the treatment of hemophilia B, a condition where the blood cannot clot properly. Part of that money will also fund the development of a gene therapy in a phase I/II trial for the treatment of the rare condition Fabry disease. The rest of the first tranche will fund the development of other gene therapies in Freeline’s pipeline, as well as expanding Freeline’s workforce and manufacturing muscle.

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There is no cure for hemophilia B at present, and gene therapies that restore the ability of the blood to clot could change the lives of patients. Freeline’s therapy consists of an adenoviral vector carrying DNA encoding factor IX, a protein that helps the blood to clot. The vector delivers the DNA into liver cells, which then have the potential to mass-produce the protein in the patient for years.

Hemophilia A and hemophilia B are the target diseases of many companies around the world. The Dutch company uniQure is one of the most advanced companies developing a hemophilia B gene therapy, with its treatment in an ongoing phase III trial.

Other companies are developing alternative methods for treating hemophilia, such as cell therapy and using molecules of RNA to block the production of anticoagulant proteins in a process called RNAi.

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