Gene Therapy for Fabry Disease

This trial tests a new gene therapy called ST-920 for individuals with Fabry disease, a rare genetic disorder that impairs the body's ability to break down certain fatty molecules. The goal is to determine if ST-920 is safe and can sustain long-term production of an important enzyme that might alleviate disease symptoms. Participants will receive a single infusion of ST-920 and undergo monitoring for a year. The trial seeks individuals diagnosed with Fabry disease who experience symptoms such as eye issues, pain in the hands or feet, reduced sweating, or skin rashes. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects in people.

The trial protocol does not specify if you must stop taking your current medications. However, if you are on systemic immunomodulatory agents, biologics, or steroids, you may need to stop as their use in the past 6 months is an exclusion criterion. Also, changes to ACE inhibitor or ARB therapy within 4 weeks prior to screening are not allowed.

Research has shown that ST-920, a gene therapy for Fabry disease, has promising safety results. Studies have observed patients who received this treatment and found it might help improve kidney function, a common problem in Fabry disease. Importantly, these studies did not find any serious safety issues, suggesting that ST-920 is generally well-tolerated in people. However, since this is an early-stage trial, researchers are still learning about its safety. Participants will be closely monitored for any side effects during the study.¹²³⁴⁵

Unlike the standard of care for Fabry Disease, which typically relies on enzyme replacement therapies, ST-920 is a gene therapy that offers a potentially transformative approach. It works by delivering a functional copy of the GLA gene directly into the patient's cells, aiming to enable the body to produce its own alpha-Gal A enzyme. This approach could eliminate the need for regular enzyme infusions and provide a long-term solution by addressing the underlying genetic cause of the disease. Researchers are particularly excited about its potential to improve outcomes across various patient groups, including those with different antibody statuses and organ involvement.

Research has shown that ST-920, a gene therapy under investigation in this trial, could help treat Fabry disease. In studies, patients who received ST-920 demonstrated improved kidney function and stable heart health, suggesting the treatment might manage some serious issues caused by Fabry disease. This trial includes different cohorts, such as those with varying doses and specific health conditions, to evaluate the therapy's effectiveness. The therapy uses a carrier to produce α-Gal A, an enzyme that helps remove harmful substances from the body. Early results have been positive, indicating potential long-term benefits.¹³⁶⁷⁸