Phase I/II clinical study exploring the potential of ST-920 investigational gene therapy to treat Fabry disease: recruiting participants now

Fabry disease is caused by changes in a gene called GLA leading to the shortage of a protein called alpha-galactosidase A (α-Gal A). Isaralgagene civaparvovec, or ST-920, is an investigational gene therapy containing the healthy copy of the GLA gene and is intended to be administered as a one-time IV infusion to restore α-Gal A.  When ST-920 is administered, it aims to deliver a healthy copy of the GLA gene to the liver, where liver cells are expected to produce the α-Gal A enzyme and secrete it via the blood stream to the rest of the body.

The STAAR Phase I/II clinical study is designed to investigate the safety and tolerability of ST-920. The study is recruiting adults aged 18 and over who have been diagnosed with Fabry disease. Several participants have  already received the study medication, and participants are now being enrolled in the selected dose group.

Preliminary clinical data were presented at the ESGCT Annual Congress 2022 on October 12, 2022 (first author: Dr. Patrick Deegan, Senior Visiting Research Fellow at the Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK) and at the NORD Breakthrough Summit on October 17-18, 2022 (first author: Dr. Jaya Ganesh, Associate Professor in the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York). Both presentations are available on the Science > Presentations page of Sangamo’s website (*.

Seventeen clinical sites are currently active in the United States, the United Kingdom, Canada, Australia, and Italy, with additional sites pending to be activated in these countries as well as in Germany and Taiwan.

Visit the STAAR Study website ( where you can see if you qualify and find a study site near you.  You can also discuss this further with the study team, who are more than happy to help.

Contact details:

* These presentations are provided as general information only and not as a prediction of any potential participant’s outcome or results from the study.