BALTIMORE — Four patients with Gaucher disease were able to stop their regular treatments after receiving a one-time infusion of a gene therapy developed by Freeline Therapeutics.
The four patients have maintained or seen improvements in certain biomarkers of Gaucher disease following the gene therapy, according to new results shared Thursday morning by Freeline at the American Society of Gene & Cell Therapy annual meeting. None of the four have had to restart either enzyme replacement therapy or substrate reduction therapy, which are standard treatments for the disease.
The data are still early: The first patient on the trial has nearly nine months of follow-up data as of the April cutoff, and stopped enzyme replacement therapy roughly two and a half months after receiving the gene therapy. So far, the company has dosed six patients in its Phase 1 GALILEO-1 study.
Preliminary imaging data also suggested that the therapy could help bone marrow health, which is notable because even in patients who receive standard treatments, diseased cells can cause damage. For one patient, the company reported improvements in fatigue as well.
People with Gaucher disease lack a key enzyme that is needed to break down a type of fat, so those fat cells build up in various parts of the body. People with the disease may have enlarged livers or spleens, low blood cell counts, and bone pain and fracturing.
Freeline’s gene therapy FLT201 delivers the gene for that crucial enzyme through a virus package called an AAV.
Syncona acquired Freeline in February for roughly $25 million earlier this year, taking the gene therapy company private. The move came after Freeline stopped work on its bleeding disorder programs.
Freeline also reported at the confab that the Gaucher therapy led to reductions or maintenance in levels of glucosylsphingosine, also known as lyso-Gb1, a molecule that builds up in patients with the disease. It also resulted in stable hemoglobin levels following withdrawal of enzyme replacement therapy or substrate reduction therapy.
Pamela Foulds“We’ve got patients who had been on therapy for many, many years and had some response with those therapies, but really still had some lyso-Gb1 still present, meaning they still had some toxic substrate in their system,” Freeline’s chief medical officer Pamela Foulds told Endpoints News.
“What was really exciting about our data is you see this really significant and fairly rapid decline to near normal levels of that lyso-Gb1,” she said.
The company also noted that there were no serious safety events following treatment. “Modest” increases in liver enzymes were observed in some patients, who were treated with immune therapy, Freeline said.
The dose that Freeline used for its Gaucher therapy was very low. While the study was designed as a dose escalation trial, Foulds said neither the company nor its independent safety review board have “felt the need for us to move up or move down on dose.”
Freeline is planning to begin a pivotal study of the gene therapy in 2025, Foulds said.