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01-Feb-2019

Freeline Announces New Program for Gaucher Disease and Poster Presentations at the 15th Annual WORLD Symposium 2019 in Orlando, USA

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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Last Updated: 01-Feb-2019