New investigational synthetic biotic for treatment of homocystinuria

Synlogic and Ginkgo Bioworks have nominated SYNB1353, an investigational synthetic biotic medicine for the treatment of homocystinuria (HCU).  

SYNB1353 is the first product developed through a research collaboration between Synlogic and Ginkgo and the first investigational medicine developed on Ginkgo’s platform to enter IND-enabling studies. Synlogic expects to file an investigational new drug (IND) application with the U.S. Food and Drug Administration (FDA) for SYNB1353 and begin clinical development in 2022. 

“SYNB1353 is an exciting addition to the Synlogic pipeline aimed at helping patients living with diseases of methionine metabolism. Starting with HCU, we see these diseases as the natural next step in our metabolic portfolio after the successful proof of concept of synthetic biotics in phenylketonuria announced earlier this year. This program builds on what we have learned from our metabolic program work to date, with the potential to provide a new therapeutic option to reduce the devastating consequences of these diseases,” said Dr. David Hava, Chief Scientific officer at Synlogic. “The advancement of SYNB1353 illustrates both the potential of our Synthetic Biotic platform to generate new therapeutic candidates and the value of our collaboration with Ginkgo.” 

Patrick Boyle, Head of Codebase at Ginkgo, said: “This is just the beginning for a new generation of medicines unlocked by the power of synthetic biology, and we’re excited to be working with Synlogic on multiple additional preclinical programs in their portfolio.”  

HCU is an inherited disorder caused by the loss of function of cystathionine beta-synthase, which results in excessive accumulation of homocysteine and its metabolites in the blood and urine. Patients develop multisystem clinical manifestations, including ectopia lentis, bone defects, intellectual disability, and life-threatening thromboembolisms. Many patients are required to comply with a rigid methionine-restricted diet and have few treatment options available. 

SYNB1353 is an engineered strain of the probiotic bacteria E. coli Nissle (EcN) which consumes methionine within the gastrointestinal tract, preventing methionine absorption and conversion to homocysteine in plasma.  

Image credit: Synlogic

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