TIDMSYNC
RNS Number : 5209S
Syncona Limited
06 November 2019
Syncona Limited
Freeline presents pre-clinical data at ASH annual meeting
06 November 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, will
report pre-clinical data on its gene therapy programme for Gaucher
Disease and Haemophilia A at the 61st American Society of
Hematology (ASH) in Orlando, Florida, 7-10 December 2019.
The announcement can be accessed on Freeline's website at:
http://www.freelinetx.com and full text of the announcement from
Freeline is contained below.
[ENDS]
Enquiries
Syncona Ltd
Siobhan Weaver / Annabel Clay
Tel: +44 (0) 20 3981 7940
FTI Consulting
Brett Pollard / Ben Atwell / Natalie Garland-Collins
Tel: +44 (0) 20 3727 1000
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 to present data on AAV-based gene therapies for Gaucher
Disease and Haemophilia A at the 61(st) ASH Annual Meeting
London, 6 November 2019 - Freeline, a clinical stage gene
therapy company focused on developing life-changing curative gene
therapies for chronic systemic diseases, will present preclinical
data on its gene therapy programme for Gaucher Disease and
Haemophilia A at the 61(st) American Society of Hematology (ASH) in
Orlando, Florida, 7-10 December 2019.
Gaucher Disease gene therapy program, FLT200
This programme is a liver-directed Adeno-associated virus (AAV)
gene therapy for the treatment of type 1 Gaucher Disease and is
designed to increase levels of the enzymes beta-glucocerebrosidase
(GCase) in the macrophages of Gaucher Disease patients. The data to
be presented indicates that in our preclinical model, a single
administration of an optimised liver directed GBA AAV vector
results in sustained elevation of the GCase enzyme in the
bloodstream and a higher level of GCase bioavailability for uptake
into macrophages in the target tissues. It is expected that the
normal GBA gene provided to the liver by FLT200 will be able to
produce a fully functioning GCase enzyme and prevent the
accumulation of the fat molecules, glucocerebroside, to toxic
levels inside the cells affected by the disease. These observations
support further development of Freeline's AAV gene therapy for
Gaucher Disease.
ABSTRACT TITLE: Liver-Directed AAV Gene Therapy for Gaucher
Disease
PRESENTER: Carlos Miranda, Principal Scientist, Freeline
DATE: Sunday, 8 December, 2019 (6:00 pm - 8:00 pm)
LOCATION: Orange County Convention Center, Hall B
Haemophilia A gene therapy program, FLT210
This programme, as all other Freeline disclosed programmes,
leverages the company's platform technology, including its
proprietary AAV capsid, known as AAVS3. Freeline's goal with the
Haemophilia A programme is to improve on existing gene therapies
for this condition by creating a novel, proprietary Factor VIII
construct that fits within the natural capacity of an AAV capsid,
which is 4.7kbp. Freeline will show data on multiple highly potent
candidates with construct sizes less than 4.9kbp, some being less
than 4.75kbp in length. The company has already selected an
optimised candidate for further development. This candidate,
designated as FLT210, is anticipated to enter IND-enabling studies
soon.
ABSTRACT TITLE: Design and Characterization of FLT210, a Potent
Next Generation AAV-hFVIII Vector Candidate
PRESENTER: Romuald Corbau, Senior Vice President Research,
Freeline
DATE: Monday, 9 December, 2019 (6:00 pm - 8:00 pm)
LOCATION: Orange County Convention Center, Hall B
"These data in Gaucher Disease and Haemophilia A are very
promising and provide further validation to our proprietary gene
therapy platform," said Chris Hollowood, Executive Chairman of
Freeline. "Freeline continues to bring potential curative therapies
to patients in our clinical phase programmes in Haemophilia B and
Fabry Disease and we expect to announce additional data from these
programmes in 2020."
- 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 Gaucher Disease
Gaucher Disease is an inherited metabolic disorder,
characterized by the build-up of glucocerebroside (GlcCer) in
lysosomes throughout the body. It is caused by mutation of the GBA1
gene, which impairs the production of the enzyme glucocerebrosidase
(GCase). Manifestations are variable and Gaucher Disease is divided
into three subtypes, of which Freeline is targeting Type 1, the
most common and non-neuronopathic form of the disease.
The condition ranges from mild to severe and may appear anytime
from childhood to adulthood. Major signs and symptoms include
enlargement of the liver and spleen (hepatosplenomegaly), a low
number of red blood cells (anemia), easy bruising caused by a
decrease in blood platelets (thrombocytopenia), lung disease, and
bone abnormalities such as bone pain, fractures, and arthritis.
Current treatment consists of enzyme replacement therapy and
substrate reduction therapy to reduce the load on the enzyme and
the accumulation of glucocerebroside.
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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