To determine the safety and efficacy of experimental gene therapy ST-920, researchers first examined the impact of the drug on mouse models of Fabry disease. As reported in Fabry Disease News, the preclinical trials ultimately showed promise. As ST-920 was able to increase alpha-GalA levels, thus preventing the accumulation of globotriaosylceramide, researchers are now looking to explore the impact of the therapy on 30 humans in a Phase 1/2 clinical trial. You can check out the researchers’ full findings published in Molecular Therapy: Methods & Clinical Development.

ST-920

Developed by Sangamo Therapeutics, ST-920 is designed to deliver a functional GLA gene to the liver, releasing alpha-GalA into the blood and reducing fat accumulation. Patients only require one dose of ST-920. In comparison to current treatments like enzyme replacement therapy, which require lifelong enzyme infusions, ST-920 offers an easier, more efficient, and more accessible treatment options for patients.

To test ST-920, researchers used mouse models of Fabry disease. Over a 6 month period, researchers intravenously injected ST-920 into some mouse models. Compared to untreated and control mice, treated mice experienced higher levels of GLA DNA and alpha-GalA. Additionally, these levels continued to increase over time, showing a sustained response. Levels of dangerous fat were reduced by up to 80% in the heart and kidneys. Within 85 days of treatment, alpha-GalA levels were, on average, 408 times higher than levels found in untreated mice. Additionally, ST-920 was safe and well-tolerated. Moving in, researchers will now test these findings in humans.

Fabry Disease

Fabry disease is a genetic lysosomal storage disorder characterized by globotriaosylceramide accumulation. Our bodies are unable to process this fat. So when the fat begins to accumulate, usually in childhood, it puts strain on the body. GLA gene mutations cause Fabry disease. It affects one in 40-60,000 males, but usually does not affect females. In some states, doctors screen for Fabry disease at birth as part of a newborn screening panel. However, this is not true for all states, so speak to your doctors. Without treatment, symptoms can lead to life-threatening complications, such as heart attack, kidney failure, or stroke. Symptoms include:

• Gastrointestinal distress
• Hearing loss
• Tinnitus
• Inability to sweat
• Red spots under the skin
• Recurrent hand and foot pain
• Cloudy or blurry vision

Learn more about Fabry disease.