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.
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