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Freeline to present data on AAV-based gene therapies for Gaucher Disease and Haemophilia A at the 61st 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 61st 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.

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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Last Updated: 07-Nov-2019