Gaucher Disease Type 1 Gene Therapy Trial Proceeds to Dose Second Cohort


The company announced that it had completed dosing in the first cohort of the GALILEO-1 trial of FLT201.

Freeline Therapeutics has completed dosing in the first cohort and dosed the first patient in the second cohort of its phase 1/2 GALILEO-1 trial (NCT05324943) evaluating FLT201 adeno-associated virus (AAV) vector gene therapy for patients with Gaucher disease type 1. The company expects to report initial clinical data in the third quarter of 2023.1

“FLT201 is a potential first- and best-in-class gene therapy for Gaucher disease Type 1, the most common type of the disease,” Michael Parini, chief executive officer, Freeline, said in a statement.1 “Advancing the program is our top strategic priority, and we are extremely pleased with our recent progress and the momentum in the trial. The completion of dosing in the first cohort of our GALILEO-1 trial of FLT201 represents a significant milestone for the program, for Freeline and for the Gaucher community. We believe FLT201 has the opportunity to dramatically reduce the disease and treatment burden for people with Gaucher disease. We look forward to reporting initial clinical data in the third quarter of this year.”

GALILEO-1 is a first-in-human, multicenter,international dose-finding study assessing the safety, tolerability, and efficacy of a single dose of FLT201 delivered intravenously. The study’s primary outcome is assessing treatment-related adverse events in the trial. FLT201 is designed to yield durable increases in glucocerebrosidase (GCase) and reduce the accumulation of harmful lipids including glucocerebroside to stop disease progression and improve outcomes. The therapy uses an AAVS3 capsid to deliver a novel GCase transgene into liver cells. Freeline also recently announced its research program in Glucosylceramidase Beta 1-linked Parkinson disease (PD) using the same engineered GCase transgene to develop a gene therapy for this population.

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“In the second quarter, we also unveiled our lead research program in GBA1-linked PD, which leverages the same longer-acting GCase variant as FLT201,” Parini added.1 “As in Gaucher disease, GBA1 mutations lead to a deficiency of the GCase enzyme and an accumulation of pathological substrates. Approximately 200,000 people with PD in the United States, United Kingdom, and 4 major European markets have GBA1 mutations, making it the most common genetic risk factor for the disease. Our GBA1-linked PD program is a natural extension of our work in Gaucher and an opportunity to extend the therapeutic potential of our longer-acting GCase variant into a genetically defined patient population with a serious unmet need.”

FLT201 is Freeline's only candidate in clinical trials after the company announced it was deprioritizing its gene therapy program FLT190 for Fabry disease in April 2023, following in the tracks of its other deprioritized gene therapy program FLT180a for hemophilia B.2,3

“While we remain encouraged by the data on FLT190 in Fabry disease, we have paused its development and are further streamlining the organization to extend our cash runway and focus on FLT201 in Gaucher disease,” Parini said in a statement at that time.“I want to extend my sincere gratitude to all of our colleagues for their dedication and their contributions to Freeline, as well as to the investigators and patients who have participated in and supported the development of FLT190.”

1. Freeline reports second quarter 2023 financial results and business highlights. News release. August 15, 2023.
2. Freeline Reports Full Year 2022 Financial Results and Provides Corporate Update. News release. April 4, 2023.
3. Freeline Reports Third Quarter 2022 Financial Results and Corporate Update. News release. Freeline Therapeutics. November 15, 2022.
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