Syncona Limited Freeline provides 12 month Haemophilia B data

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Syncona Limited

08 July 2019

Syncona Limited

Freeline provides 12 month Haemophilia B data at ISTH

8 July 2019

Syncona Ltd, a leading healthcare company focused on founding, building and funding global leaders in life science, notes the announcement that its portfolio company, Freeline Therapeutics, has presented 52-week data from the first cohort of two patients in the ongoing B-AMAZE FLT180a Phase 1/2 trial in Haemophilia B at the International Society on Thrombosis and Haemostasis (ISTH) congress in Melbourne, Australia, 6-10 July 2019.

The full text announcement is contained below and can be accessed on Freeline's website at: http://www.freelinetx.com

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Enquiries

Syncona Ltd

Siobhan Weaver / Annabel Clay

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FTI Consulting

Brett Pollard / Natalie Garland-Collins

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Copies of this press release and other corporate information can be found on the company website at: www.synconaltd.com

About Syncona

Syncona is a leading FTSE250 healthcare company focused on founding, building and funding global leaders in life science. Our vision is to deliver transformational treatments to patients in truly innovative areas of healthcare while generating superior returns for shareholders.

We seek to partner with the best, brightest and most ambitious minds in science to build globally competitive businesses.

We take a long-term view, underpinned by a deep pool of capital, and are established leaders in gene and cell therapy. We focus on delivering dramatic efficacy for patients in areas of high unmet need.

Freeline announces updated Factor IX data and expansion of its Haemophilia pipeline

- Freeline presents twelve-month clinical data for first cohort of two patients in FLT180a, an AAV gene therapy for Haemophilia B, at ISTH -

- Announces a new programme in Haemophilia A -

London, 8 July 2019 - Freeline, a biotechnology company focused on developing curative gene therapies for chronic systemic diseases, presented long-term FLT180a data at the International Society on Thrombosis and Haemostasis (ISTH) congress in Melbourne Australia, 6-10 July 2019. In addition, the Company is pleased to announce that it is broadening its pipeline with a new preclinical programme, FLT210, to target Haemophilia A, a bleeding disorder caused by a deficiency in clotting factor VIII.

New programme in Haemophilia A:

The new Freeline Haemophilia A programme leverages the company's platform technology, including its proprietary adeno-associated viral (AAV) capsid, known as AAVS3. The programme is based on a proprietary approach and has yielded promising preclinical data which will be presented at a future scientific forum. By incorporating all aspects of the Freeline platform, the programme is designed to provide high and consistent expression at a relatively low dose. FLT210 will soon enter Investigational New Drug (IND) enabling studies.

Twelve-month clinical data update in Haemophilia B:

The Freeline Haemophilia B programme, FLT180a, uses the AAVS3 capsid and a gain of function variant of human factor IX (FIX). The therapy is in a Phase 1/2 trial known as B-AMAZE, with the goal of normalising FIX activity in patients with moderate and severe Haemophilia B. At ISTH Freeline presented data from the first cohort of two patients who were treated with the lowest study dose (4.5x10(11) vg/kg). Over the 12 months since infusion both patients' liver enzymes remained within the normal range at all time points and the patients' transgene expression was stable, with Factor IX levels of 40.5+/-4.5% at week 52, assessed by the one stage clotting assay using the SynthASil(TM) reagent. Following treatment, both patients were free of spontaneous bleeding episodes and did not require any Factor IX supplementation.

Freeline continues to enrol patients in the B-AMAZE trial, with the goal of optimising the dose to express Factor IX in the normal range of 50-150%, which would free patients from the symptoms of the disease.

Chris Hollowood, Executive Chairman of Freeline said:

"We are pleased to report updated data on the first two patients in our Haemophilia B trial. The results provide early validation for the potential durability and stability of our therapy at the lowest dose cohort. We continue to progress dose optimisation in additional patients and look forward to providing a further update as we seek to develop a functional cure for people with Haemophilia B."

"We believe there is a significant opportunity for Freeline's highly efficient capsid and manufacturing platform to offer gene therapies that substantially improve patients' lives in a wide range of chronic systemic diseases, and we are seeking to leverage this same platform across Haemophilia A and B, Fabry and Gaucher Diseases, and ultimately into non-monogenic disorders."

Poster & presentation for ISTH Congress were as follows:

Presenter: Jonathan Foley

Session: Poster Networking Session

Abstract title: FLT180a: Next Generation AAV Vector for Haemophilia B - Long Term, Follow-up and

In-depth Analysis of Transgenic FIX Using One-stage, Chromogenic and Global Assays

Date: Sunday, 7 July 2019 from 6:30 - 7:30 pm

Location: Exhibition hall

Presenter: Amit Nathwani

Session: State-of-the-Art Session. ISTH-WFH Joint Session: Haemophilia Management

Presentation title: Gene Therapy

Date: Monday, 8 July 2019 from 8:00 - 8:25 am

Location: Plenary Hall SOA 08

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Further information:

JW Communications

Julia Wilson

[phone]

[e-mail]

About Freeline

Freeline is a privately-held clinical-stage biotechnology company focused on AAV based gene therapy targeting the liver. Our vision is to create better lives for people suffering from chronic systemic diseases using the potential of gene therapy as a one-time curative treatment. Freeline is headquartered in the UK and has operations in Germany and the US.

About Haemophilia

Haemophilia is a genetic bleeding disorder where a protein made by the body to help make blood clot is either partly or completely missing. This protein is called a clotting factor. With Haemophilia A there is a deficiency of clotting factor VIII (eight) and with Haemophilia B there is a deficiency of clotting factor IX (nine). Haemophilia mainly affects boys and men; women can be 'carriers' of the affected gene and may experience symptoms. Haemophilia A is the most common type of Haemophilia affecting about one in every 10,000 males, while Haemophilia B affects about one in every 50,000 males. Haemophilia is classed as mild, moderate or severe, depending on the level of clotting factor VIII or IX in the blood and is diagnosed through blood tests.

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July 08, 2019 02:00 ET (06:00 GMT)