NEW YORK – Freeline Therapeutics announced Monday that the first patient with Gaucher disease type 1 has received its investigational gene therapy FLT201 in a Phase I/II trial.
The GALILEO-1 trial is a first-in-human dose escalation study assessing the safety, toxicity, and efficacy of a single dose of FLT201 in up to four cohorts of adult patients with Gaucher disease type 1. In this study, Freeline is also hoping to identify the FLT201 dose it will advance into a Phase III trial.
Gaucher disease is an inherited condition caused by a mutation in the GBA1 gene that results in low levels of glucocerebrosidase (GCase). This in turn allows fatty substances to accumulate in organs, particularly in the spleen and liver, leading to low blood counts, bone pain, and reduced lung function. Freeline is focused on studying the most common form of the disease, type 1.
FLT201 uses an adeno-associated virus S3 capsid to deliver a functional version of the GBA1 gene. It is designed to enter liver cells and generate durable increases in GCase.
Hertfordshire, UK-based Freeline expects to report initial data on safety and enzyme activity from the first cohort in the GALILEO-1 trial in Q3 of this year. "The dosing of the first patient in the GALILEO-1 trial of FLT201 marks an important step forward for the Gaucher community," Ozlem Goker-Alpan, founder and CEO of the Lysosomal and Rare Disorder Research and Treatment Center and an investigator in the study, said in a statement.
"Existing therapies come with a heavy lifelong treatment burden, and many patients continue to experience serious symptoms," she added. "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."
Freeline on Monday also unveiled plans to develop a gene therapy for GBA1-linked Parkinson's disease. The company aims to leverage the same transgene in FLT201 to design a gene therapy for the 5 percent to 15 percent of Parkinson's patients with GBA1 mutations. Patients with these mutations are at increased risk of developing earlier onset disease, having more severe symptoms, and progressing to dementia.
No disease-modifying therapies currently exist for Parkinson's disease, and current treatments tend to grow less effective over time. "Our GBA1-linked Parkinson’s disease program is a natural extension of our work in Gaucher disease 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," Freeline CEO Michael Parini said in a statement.