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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
[ENDS]
Enquiries
Syncona Ltd
Siobhan Weaver / Annabel Clay
Tel : +44 (0) 20 3981 7940
FTI Consulting
Brett Pollard / Natalie Garland-Collins
Tel: +44 (0) 20 3727 1000
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
- Ends -
Further information:
JW Communications
Julia Wilson
+44 (0) 7818 430877
juliawilsonuk@gmail.com
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.
This information is provided by RNS, the news service of the
London Stock Exchange. RNS is approved by the Financial Conduct
Authority to act as a Primary Information Provider in the United
Kingdom. Terms and conditions relating to the use and distribution
of this information may apply. For further information, please
contact rns@lseg.com or visit www.rns.com.
END
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