Sangamo Therapeutics, with its primary focus on genomic medicine, recently issued a news release announcing data from its phase 1/2 STARR trial. The data evaluated isaralgagene civaparvovec, or (ST-920) as a treatment for Fabry disease, a multisystem disorder.
The STARR trial showed sustained elevated α-Gal A Enzyme Activity in Fabry Disease trial participants. The a-Gal A enzymatic activity was evaluated through the dried blood spots of male patients diagnosed with Fabry disease. The rates ranged from 0 to 1.7 μmol/h/L compared to 4.2-17.3 μmol/h/L in healthy people who were not Fabry disease patients.
In addition to dried blood spots, a-Gal A can be evaluated in leukocytes, plasma, and cultured fibroblasts. However, female patients who have inherited a specific gene from each of their parents exhibited enzymatic activity of a-Gal A with a range in dried blood spots of 0-12.6 μmol/h/L. These numbers are comparable to those of individuals who do not have Fabry disorder. Therefore, genetic testing may be used in males but it is less reliable in diagnosing Fabry disorder in females.
The cutoff date was July 21, 2022, at which time the treatment was still well tolerated without serious adverse events. All nine patients involved in dose escalation showed elevated a-Gal A activity. As of the last measurement, the nine patients showed a-Gal A activity of between two to thirty fold.
Currently, the STAAR study Phase ½ is in the expansion stage with four participants. This cohort includes the first female participant.
The study data was presented this month at the European Society of Gene & Cell Therapy’s 29th Congress.
About Sangamo Therapeutic
Sangamo Therapeutics, a biopharmaceutical company is focused on developing novel genomic medicines for patients suffering from diseases that lack treatment options.
Details of each cohort and α-Gal A patient activity are available here.