Freeline, a biotechnology company focused on developing curative gene therapy for chronic systemic diseases, today announces a new program for Gaucher Disease, an inherited metabolic disorder characterized by the progressive build-up of glucocerebroside in lysosomes throughout the body
London, February 1, 2019 – Freeline, a biotechnology company focused on developing curative gene therapy for chronic systemic diseases, today announces a new program for Gaucher Disease, an inherited metabolic disorder characterized by the progressive build-up of glucocerebroside in lysosomes throughout the body. Freeline is targeting Type 1 Gaucher Disease, the most common and non-neuronopathic form of the disease.
In addition, Freeline announces that it will present two posters at the 15th Annual WORLD Symposium, to be held February 4-8, 2019 in Orlando, Florida. The first poster presents preclinical data on the long-term follow-up of Fabry mice treated with liver-directed AAV gene therapy, and the second poster presents preclinical data evaluating liver-directed AAV gene therapy to treat Gaucher Disease.
Poster presentation details are as follows:
Fabry Disease:
ABSTRACT TITLE: Liver-directed gene therapy corrects Fabry disease in mice
PRESENTER: Jey Jeyakumar
DATE: Tuesday, February 5, 2019 from 4:30-6:30 PM
LOCATION: Regency Ballroom R, Hyatt Regency Orlando
Gaucher Disease:
ABSTRACT TITLE: Liver-directed AAV gene therapy to treat Gaucher disease
PRESENTER: Carlos Miranda
DATE: Wednesday, February 6, 2019 from 4:30-6:30 PM
LOCATION: Regency Ballroom R, Hyatt Regency Orlando
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Further information:
JW Communications
Julia Wilson
+44 (0) 7818 430877
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 Fabry Disease
Fabry Disease is an inherited, X-linked disease characterized by the progressive accumulation of glycosphingolipids in lysosomes throughout the body. It is caused by mutations in the gene encoding of the α-galactosidase A enzyme (GLA) responsible for the breakdown of globotriaosylceramide (Gb3), a type of glycosphingolipid.
The condition ranges from mild to severe and may appear anytime from childhood to adulthood. The progressive accumulation of Gb3 leads to organ damage, major disability and often early mortality.
Symptoms and signs include neuropathic pain, impaired sweating, gastrointestinal symptoms, renal failure, heart disease and increased risk of stroke. Current treatment consists of enzyme replacement therapy and chaperone therapy to temporarily clear Gb3 accumulation and alleviate symptoms.
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.
