Wellcome invests $38M in another gene therapy startup, targeting hemophilia

The Wellcome Trust's investment arm is putting up £25 million ($38 million) to launch a new company developing a potential one-time treatment for hemophilia and planning to build a pipeline of gene therapies.

Dubbed Freeline Therapeutics, the company is getting off the ground with help from Wellcome's Syncona subsidiary and technology licensed from University College London, looking to build the clinical case for a treatment that could reverse hemophilia B.

Hemophilia, which can lead to fatal bleeding episodes, results from a deficiency of key proteins needed for coagulation. Freeline's top prospect uses a harmless strain of adeno-associated virus to correct that deficiency, delivering a healthy copy of the gene responsible for regulating one such protein and treating hemophilia in the process.

UCL's Amit Nathwani

The biotech's in-licensed candidate, developed by UCL professor Amit Nathwani, reduced bleeding episodes by 90% over three years in a 10-patient trial, spurring the production of coagulation proteins with no toxic effects. Those results, published in The New England Journal of Medicine, form the bedrock of Freeline's clinical ambitions.

"Building on the success of that study, we partnered with Syncona to form Freeline to provide an opportunity to realize our ambition of bringing multiple therapeutics to the patients that need them," Nathwani said in a statement. "Freeline will bring industry-leading development people and our expertise together to ensure our clinical translation is rapidly converted to registered therapeutics."

Syncona partner Christian Groendahl is stepping in to serve as CEO of the company, and Nathwani is joining as chief scientific officer. Freeline's first priority is hemophilia, but the company believes Nathwani's viral platform has potential in other, unnamed diseases as well.

Dimension Therapeutics ($DMTX), which went public this year, is taking a similar approach to bleeding disorders, planning to get its gene therapy for hemophilia B into clinical trials this year and working with Bayer on an early-stage treatment for hemophilia A.

Freeline is Syncona's second big bet on gene therapy this year, following the firm's investment in an University of Oxford spinout called NightstaRx that is at work on a one-time treatment for a rare disease that leads to blindness.

- read the statement