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Spin-Off Developing Gene Therapies for Blood Disorders

Red blood cells

Red blood cells illustration (geralt, Pixabay)

11 December 2015. A new spin-off company from University College London in the U.K. is developing gene therapies to treat hemophilia and related diseases. The company, Freeline Therapeutics, is raising £25 million ($US 38 million) in its first venture funding round.

Freeline Therapeutics is founded and commercializing research by UCL hematology professor Amit Nathwani, who discovered and tests gene-therapy treatments for hemophilia B, an inherited disorder. Hemophilia B results from the absence of a clotting protein in blood known as factor IX. People with hemophilia B bleed longer than people with factor IX, with some 60 percent of cases considered severe, meaning people with the condition experience bleeding after an injury, including spontaneous bleeding into muscles and joints.

The treatments designed by Nathwani and colleagues transfer a healthy gene, using adeno-associated viruses, naturally occurring microbes that can infect cells, but do not integrate with the cell’s genome nor cause disease. The healthy gene then expresses proteins that produce the missing factor IX. In addition, Nathwani’s method avoids liver toxicity experienced in some other gene therapies.

Nathwani is testing the efficacy and dosage levels of this gene therapy in an intermediate-stage clinical trial with children having severe cases of hemophilia B at St. Jude’s Children’s Research Hospital in Memphis. In results published last November in New England Journal of Medicine, Nathwani and colleagues found a single dose of the therapy provided factor IX expression in the 10 participants for as long as three years, allowing them to stop factor IX replacement regimens and in some cases even take part in sports. The main adverse effect was a rise in ALT enzymes indicating liver damage, which were treated successfully with prednisolone, a drug for inflammation.

Freeline Therapeutics is receiving £25 million in its first venture round from Syncona LLP, a subsidiary of the Wellcome Trust, a global health charitable foundation. Syncona makes evergreen investments that provide sustained capital over longer terms rather than a single up-front cash infusion. Syncona’s portfolio includes other gene-therapy companies, as well as enterprises in cell therapies and diagnostics.

Syncona is also providing Freeline’s top management, with partners Christian Groendahl and Chris Hollowood joining Freeline Therapeutics as CEO and chairman respectively. Nathwani will serve as Freeline’s chief scientist.

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Hat tip: Fierce Biotech

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