UCL spinout commercialises gene therapy research to progress new treatments for haemophilia B

UCL spinout Freeline Therapeutics is taking groundbreaking gene research from UCL, with the aim of developing it into a potentially life-changing treatment for people with haemophilia B.

Haemophilia B is a rare and inherited bleeding disorder caused by low levels of a particular protein in the blood called factor IX (or FIX for short). FIX is needed for forming clots, and patients currently have to inject themselves with an FIX replacement, usually weekly, to prevent excessive bleeding.

Despite advances in treatment, many patients experience debilitating joint damage due to bleeding, and the disease can have a serious adverse impact on their lives.

But now, thanks to research developed at UCL, and subsequently spun out as Freeline Therapeutics, there could be new hope for people with haemophilia B.

If approved, Freeline’s gene therapy could provide lasting benefits to these haemophiliac patients. The therapy would require just a single treatment to alter a patient’s FIX levels, doing away with the need for regular injections in many patients.

Freeline co-founder Professor Amit Nathwani (UCL Cancer Institute) explains: “Gene therapy works essentially by allowing us to introduce a synthesised gene into a patient’s body, to compensate for one that’s not working properly, as is the case in those with haemophilia. Functional FIX is then released into the blood, which reduces the risk of excessive bleeding.”

Translating innovation into potentially life-changing technologies

This groundbreaking new approach attracted the interest of biotech investor Syncona in 2015. With help from UCL Business (UCLB), the commercialisation company of UCL (and part of UCL Innovation & Enterprise), the team went onto launch Freeline.

The spinout is now developing three different gene therapy candidates, to transform Professor Nathwani’s research into potentially life-changing treatments.

Professor Nathwani explained: "In nine out of the ten haemophilia B patients we tested this on, in our Phase 1/2 B-AMAZE clinical trial, the treatment led to a sustained increase in FIX protein production, which led to a decrease in excessive bleeding. They also no longer required weekly injections of FIX protein. After 26 weeks, five patients had normal levels of FIX protein, three had low but increased levels in the mild range, and just one patient had an increased level above his baseline.

“Building on our initial success, we formed Freeline to realise our ambition of bringing multiple gene therapies to patients. Commercialising the research has allowed us to combine some of the best expertise in drug development with years of groundbreaking research at UCL, to ensure that approaches that show promise in early phase trials can move rapidly into marketed therapeutics, if approved.”

Initially joining Freeline as Chief Scientific Officer, and now as Clinical and Scientific Adviser and a Director of the company, Professor Nathwani explains the advantage of doing this work in a company setting.

“Taking our research out into a spinout business means we now have a whole bunch of experts working with us who have significant experience of developing therapies in industry. They brought vital know-how that’s helping us come up with safe and effective ways of processing novel therapies into clinics.

“Thanks to the B-AMAZE trial, we have a growing body of evidence that gene therapy has the potential to free patients from the challenges of lifelong therapy, and could provide treatment for diseases where none exists today.”

Speeding up trials and treatment

Syncona invested £25 million into Freeline in 2016, alongside the UCL Technology Fund. A further $218 million was raised in subsequent fundraising rounds.  An initial public offering on Nasdaq followed in 2020, raising approximately $162 million. 

These investments have enabled the company to invest in research and development laboratories in Stevenage and advance development programmes through both pre-clinical studies and clinical trials.

Richard Fagan, Director of BioPharm at UCLB, led the creation of the company with Syncona. Richard said: “Gene therapy is a highly innovative field, in which UCL has significant expertise. We were excited to partner with Syncona to commercialise these potentially life-changing treatments, so that as many patients as possible could soon be benefitting from them.”

Links

Find out more about:

• Freeline Therapeutics
• Innovation and enterprise support for UCL staff
• UCL Business
• UCL Cancer Institute
• Professor Amit Nathwani's academic profile
• Syncona Ltd

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This case study is based on a story written in July 2022: Novel gene therapy could reduce bleeding risk for haemophilia patients.

Image credit: iStock.com/koto_feja