“FLT201 is a potential first- and best-in-class gene therapy for Gaucher disease Type 1, the most common type of the disease,” said Michael Parini, Chief Executive Officer of Freeline. “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. In the second quarter, we also unveiled our lead research program in GBA1-linked Parkinson’s disease, which leverages the same longer-acting GCase variant as FLT201,” Parini continued. “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 Parkinson’s disease in the US, UK and four major European markets have GBA1 mutations, making it the most common genetic risk factor for the disease. Our GBA1-linked Parkinson’s disease 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.”
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