Synlogic, a biotechnology company in Cambridge, Massachusetts is making progress on a potential treatment for HCU. Synlogic specializes in designing potential new drugs by engineering probiotics to treat diseases in new ways. For several years, Synlogic has been sharing their work on a potential new drug for PKU, and shared positive news for this drug last fall. As many of you know, PKU is an inborn error of metabolism, like HCU, and people with PKU are also required to live with very challenging food restrictions.

In November of 2021, Synlogic announced that the company had a new potential drug for HCU called SYNB1353, designed with a partner company called Gingko Bioworks. SYNB1353 is given orally. It is a specially designed probiotic that has been engineered to consume methionine, thereby lowering homocysteine levels in patients with HCU. A potential benefit of using probiotics as the “vehicle” for new drugs is that probiotics have been studied for more than a hundred years, and have evolved to work in the GI (gastrointestinal) tract, which is where SYNB1353 is designed to consume methionine.

In 2022, Synlogic is expected to start to study this potential treatment in healthy volunteers to assess its safety in humans. The studies in 2022 will not be in people with HCU, but the information will be important for the company in assessing studies in HCU afterwards. The Company expects to report results from these first studies before the end of the year.

Synlogic shared scientific information about SYNB1353 at the International Congress of Inborn Errors of Metabolism through a poster presentation. You can see that poster below, or by clicking here.