UK biotech Freeline Therapeutics has extended its Series C round to take its gene therapy for hemophilia B to a pivotal trial, while hinting at the possibility of an IPO in the US later this year.

This final closing adds an additional €70M to a €36M Series C investment by London-based VC Syncona in December last year. Though originally planning to double its contribution in a second tranche, Syncona has decided to stick to the initial amount due to the interest from new investors.

Danish investor Novo Holdings and US-based Eventide Asset Management and Wellington Management Company led the latest closing. 

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Freeline will primarily use the funding to progress its pipeline of gene therapies. In particular, the company will start a pivotal trial for its lead candidate for the treatment of the blood clotting disorder hemophilia B. It hasn’t specified when the trial will begin, or which clinical phase the pivotal trial will be. Freeline is currently identifying the best dose to use in a phase I/II trial. 

The company will also use some of the funds to progress an ongoing phase I/II trial testing a gene therapy for Fabry disease, a rare genetic disorder caused by lack of an enzyme called alpha-galactosidase A that normally breaks down fats in the blood. When levels are low or absent these fats can build up around the body and cause tissue damage that can result in symptoms such as cardiovascular disease and kidney problems. 

Freeline will also aim to progress other earlier-stage candidates to clinical trials. These include gene therapies for Gaucher disease, which also involves faulty fat metabolism in the blood, and hemophilia A. The company will also expand its gene therapy manufacturing capacity.

The company’s technology consists of delivering therapeutic DNA into patients via a viral vector. The technology has already been proven to be able to transfer the therapeutic DNA at a good rate into the blood via the liver. 

Notably, at the same time as it announced the closing of the Series C round, Freeline also stated that “it expects to consider additional capital raising options this year, which could include an initial public offering in the United States,” although the company chose not to comment further on this announcement. 

Freeline is by no means the only company trying to treat hemophilia using gene therapy. Several other biotechs are racing to get effective gene therapies for this genetic disorder to the market. For example, Dutch biotech uniQure recently teamed up with CSL Behring in a €1.8B deal to commercialize its own hemophilia B gene therapy and expects phase III results towards the end of the year. Also in the runnings are Pfizer and Spark, which together aim to complete a phase III trial late next year.

Hemophilia is also one of the first targets for several companies working on a novel type of gene therapy using CRISPR gene editing. US biotech Intellia Therapeutics, co-founded by CRISPR pioneer Jennifer Doudna, has a partnership with another US biotech called Regeneron to develop CRISPR-based gene therapies for both hemophilia A and B.


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