Freeline to Host Virtual KOL Event on its Gene Therapy Candidate, FLT201, in Gaucher Disease
Webinar to take place on August 1, 2023, at 8:30 AM ET
LONDON, July 25, 2023 (GLOBE NEWSWIRE) -- (Nasdaq: FRLN) today announced it will host a virtual Key Opinion Leader (KOL) event on FLT201, its adeno-associated virus (AAV) gene therapy candidate in Gaucher disease Type 1, on Tuesday, August 1, 2023 at 8:30 a.m. ET.
The event will feature Gaucher disease expert, Dr. Reena Sharma, Consultant Adult Metabolic Medicine and Honorary Senior Lecturer at the Salford Royal Hospital in the United Kingdom, who will discuss the epidemiology, treatment landscape, and unmet need in Gaucher disease.
The Freeline leadership team will provide a corporate overview and outline the clinical progress of FLT201. Currently, FLT201 is being evaluated in the GALILEO-1 study, a first-in-human, international, multicenter Phase 1/2 dose-escalation trial assessing safety, tolerability and efficacy of a single intravenous dose of FLT201, with the aim of identifying a dose for further development in a Phase 3 clinical trial. Dosing commenced in the trial last month.
A live question and answer session will follow the formal presentations. To access the event, participants may register . While not required, it is recommended that participants join 10 minutes prior to the scheduled start. A live webcast of the event will be available on the Investors section of Freeline’s website at An archived replay will be available on the website for at least 90 days.
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 lungs. Despite treatment with existing therapies, many people with Gaucher disease continue to experience symptoms and disease progression. Gaucher disease affects approximately 18,000 people in the United States, United Kingdom, France, Germany, Spain, Italy and Israel.
About FLT201
FLT201 is an adeno-associated virus (AAV) gene therapy candidate that is currently being investigated in the GALILEO-1 Phase 1/2 clinical trial in adults with Gaucher disease type 1. FLT201 is designed to generate durable increases in glucocerebrosidase (GCase) and reduce the accumulation of harmful substrates, with the aim of providing a one-time treatment that can stop disease progression, improve outcomes and free people from lifelong treatment. FLT201 uses Freeline’s proprietary AAVS3 capsid to introduce a novel transgene into liver cells to produce a rationally engineered GCase variant. In preclinical studies, the GCase variant has demonstrated a 20-fold increase in half-life at lysosomal pH conditions compared to wildtype human GCase. Preclinically, FLT201 has shown robust GCase expression, as well as significant GCase uptake and substrate reduction in key tissues.
About Freeline Therapeutics
Freeline is a clinical-stage biotechnology company focused on developing transformative gene therapies for chronic debilitating diseases. Freeline uses its proprietary, rationally designed AAV vector and capsid (AAVS3), along with novel promoters and transgenes, to deliver a functional copy of a therapeutic gene into human liver cells, thereby expressing a persistent functional level of the missing or dysfunctional protein into a patient’s bloodstream. The company is currently advancing FLT201, a highly differentiated gene therapy candidate that delivers a novel transgene, in a Phase 1/2 clinical trial in people with Gaucher disease type 1. Freeline has additional programs in research, including one focused on GBA1-linked Parkinson’s disease that leverages the same novel transgene as FLT201. Freeline is headquartered in the UK and has operations in the United States. For more information, visit or connect with Freeline on and .
Forward-Looking Statements
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Media and Investor Contact:
Naomi Aoki
Senior Vice President, Head of Investor Relations & Corporate Communications
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