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Freeline raises £88m to accelerate haemophilia gene therapy

Spin-out from London university joins fast-developing field

UK start-up Freeline Therapeutics has raised an impressive £88m ($116m) in a second-round financing that will allow it to fund additional testing of its lead haemophilia B gene therapy, and take another for Fabry disease into the clinic.

Freeline’s chief executive Anne Prener – who joined the company last July and previously led Baxalta’s haematology programmes – says the cash injection would also be used to beef up its manufacturing and analytics capabilities.

The Stevenage-based biotech has developed an adeno-associated virus (AAV) technology platform designed to deliver genes coding for therapeutic proteins into cells of the liver via an injection into the peripheral blood, and already has clinical trials in hand for its first haemophilia B candidate.

Amit Nathwani

The gene therapy has developed from research conducted by Freeline’s chief scientific officer Prof Amit Nathwani (pictured), who is also professor of haematology at University College London and director of a haemophilia centre at the Royal Free Hospital. Prof Nathwani was the first to show that bleeding could be corrected in patients with severe haemophilia B using a gene transfer approach.

Since then, several other biotechs have started to develop gene therapies for haemophilia, and Freeline will need the new funding to help it catch up with the leaders. Notable player are Spark Therapeutics – now in phase III trials with its Pfizer-partnered SPK-9001 therapy – and UniQure which is planning to start pivotal trials of its AMT-060 candidate this year. Following after is Sangamo with SB-FIX in phase I/II testing.

Baxalta (now part of Shire) was an early player in the field with its BAX 335 therapy, but has fallen behind after abandoning the phase I/II programme in 2016, turning its attentions to an early-stage platform that it felt had more chance of being commercially viable.

Haemophilia - both type A and B - is seen as a low-hanging fruit among gene therapy targets as it is caused by a single gene defect, it is relatively easy to deliver therapy to the target organ (the liver) and the effects are easily measured using well-recognised biomarkers.

“Establishing our fully integrated AAV gene therapy platform has been a key focus since the company’s inception, allowing secure and timely manufacture of products using our cutting-edge manufacturing technologies,” said Prener. “The additional funding will enable us to continue this important work.”

Sycona the driver for UK gene therapy start-ups

Playing a key role in the flowering of gene therapy companies in the UK is London-based life sciences investment fund Syncona, which is investing heavily in numerous companies. The new funding for Freeline adds to almost £35m the company raised in a Series A round, which was led by Syncona with participation from UCL Technology Fund.

Just last week Syncona announced the formation of two further gene therapy companies, bringing its portfolio to no fewer than five firms.  Last week it launched SwanBio, focused on the Central Nervous System, and Orbit Biomedical, which is based on a leading sub-retinal surgical delivery platform acquired from Janssen Biotech.

This adds to Freeline and its focus on chronic systemic disease, Nightstar ( specialising in inherited retinal diseases), and Gyroscope, which specialises in retinal inflammation. Syncona says its portfolio companies now covers every tissue compartment where gene therapy has proof of concept (retina, liver, CNS), and has also invested in the manufacturing process, which is so integral to gene and cell therapies.

Article by
Phil Taylor

19th June 2018

From: Research

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