Freeline Therapeutics Holdings plc announced that the first patient has been dosed in the Phase 1/2 GALILEO-1 clinical trial evaluating FLT201, its adeno-associated virus (AAV) gene therapy candidate, in Gaucher disease type 1. Gaucher disease is a debilitating genetic disorder in which a deficiency of the GCase enzyme leads to a buildup of fatty substances in the organs, causing symptoms including enlarged spleen and liver, low blood counts, bone pain and reduced lung function. FLT201 represents a promising approach for addressing the underlying cause of Gaucher disease with a one-time gene therapy, potentially providing a more effective and less burdensome option for patients. GALILEO-1 is a first-in-human, international, multicenter Phase 1/2 dose-escalation study.

The trial will assess safety, tolerability and efficacy of a single intravenous dose of FLT201 in up to four cohorts of escalating doses, with the aim of identifying a dose for further development in a Phase 3 clinical trial. 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. 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". All statements, other than historical facts, including statements regarding FLT201's potential to be a first- and best-in-class gene therapy, stop disease progression and improve outcomes with a one-time treatment, that the Company expects to report initial data from the first cohort of the GALILEO- 1/2 clinical trial of FLT201 in the third quarter of this year, and regarding the potential to extend the Company's GCase variant into GBA1-linked Parkinson's disease and create a life-changing gene therapy for this patient population are forward-looking statements.