A Gene Therapy Trial for Fabry Disease Has Begun Dosing Patients

According to a story from Fabry Disease News, the first patient in a clinical trial testing the investigational gene therapy FLT190 has reportedly been dosed. FLT190 is being developed as a treatment for Fabry disease, a rare genetic disorder. Enrollment for this phase 1/2 clinical trial is still ongoing. The Fabry disease patient community should follow the progress of this trial very closely, as a successful gene therapy could be significant breakthrough in the treatment of this illness.

About Fabry Disease

Fabry disease is a rare genetic disorder that primarily affects the heart, skin, and kidneys. As a lysosomal storage disease, it is characterized by a deficiency in the enzyme responsible for processing sphingolipids, which accumulate in the body as a result. The disorder is caused by mutations of the GLA gene. Symptoms include pain (which can affect the extremities, the entire body, or the digestive tract), kidney dysfunction, abnormalities of the heart valve and heart rhythms, fatigue, inability to sweat, and angiokeratomas (small red dots that appear on the skin). Treatments include enzyme replacement therapy, treatments to address organ specific problems, and Galafold. Galafold is effective in roughly 50 percent of patients, and only works for patients with certain types of mutations. Enzyme replacement therapy can help partially halt or reverse disease progression. To learn more about Fabry disease, click here.

About FLT190 and The Clinical Trial

FLT190 is being developed by Freeline, a company that is focused on the creation of gene therapies for chronic illnesses. Like most gene therapies, FLT190 utilizes an adeno-associated virus (AAV) as a mechanism for the delivery of the genetic component, which in this case is a healthy, un-mutated copy of the GLA gene. The therapy will be delivered in just a single dosage.

The study will include 12 adult males with Fabry disease that have the “classic” type 1 variant of the illness. FLT190 will be delivered via slow, intravenous infusion; patients will be initially observed for a period of nine months followed by an extended monitoring period. The trial will include patients that have been treated with other drugs before and those that have not. 

A mouse model of Fabry disease that was treated with the therapy has shown promising results.

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