FRLN - Freeline to Present New Clinical Data for FLT201 in Gaucher Disease in Oral Presentation at Upcoming ESGCT 30th Annual Congress | Benzinga
LONDON, Oct. 16, 2023 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (NASDAQ:FRLN) today announced that it will present new clinical data from its ongoing Phase 1/2 GALILEO-1 trial of FLT201, its adeno-associated virus (AAV) gene therapy candidate, in patients with Gaucher disease Type 1 at the European Society of Gene & Cell Therapy (ESGCT) 30th Annual Congress being held October 24-27, 2023 in Brussels, Belgium.
The oral presentation will include data on safety, tolerability, GCase enzyme activity, substrate, and hemoglobin and platelet levels from the first two patients treated with FLT201.
Details of the oral presentation are below:
Presentation Title: Results from GALILEO-1, a First-In-Human Clinical Trial of FLT201 Gene Therapy in Patients with Gaucher Disease Type 1
Presenter: Ozlem Goker-Alpan, MD, Lysosomal and Rare Disorders Research and Treatment Center
Date and Time: Wednesday, October 25 from 14:30-16:30 CEST (8:30-10:30 a.m. ET)
Session: 5B – Gene Therapy for Metabolic Diseases at Clinical Stage
Additional details on the meeting can be found at the ESGCT 30th Annual Congress website. The presentation materials will be available on the Investors section of Freeline's website following presentation at the ESGCT Congress.
About Gaucher Disease
Gaucher disease is caused by a mutation in the GBA1 gene that results in abnormally low levels of glucocerebrosidase (GCase), an enzyme needed to metabolize a certain type of lipid. As a result, harmful substrates glucosylceramide (Gb-1) and glucosylsphingosine (lyso-Gb1) build up in cells that then accumulate in various organs, causing inflammation and dysfunction. Gaucher disease is hereditary and presents in various subtypes. Freeline is currently focused on Gaucher disease Type 1, the most common form of the disease, which affects the health of the spleen, liver, bone and lung. Despite treatment with existing therapies, many people with Gaucher disease continue to experience symptoms and disease progression. Gaucher disease affects approximately ...