Investigational Synthetic Biotic Medicine for the Treatment of Homocystinuria

Homocystinuria (HCU) is an inherited disorder that affects tens of thousands of people every day. HCU occurs when a person is unable to metabolize the amino acid methionine, a precursor to homocysteine; the result is an excessive accumulation of homocysteine and its metabolites in a person’s blood and urine. Patients develop multiple symptoms, including bone defects, intellectual disabilities, and life-threatening blood vessel obstructions. Many patients are required to comply with rigid, restrictive diets and have few treatment options available.

What if we could tell bacteria inside these patients to metabolize methionine, lowering homocysteine levels and reducing the effects of diseases like HCU?

SYNB1353, an engineered strain of the probiotic bacteria E. coli Nissle (EcN), was designed and is being developed through a collaboration between Synlogic and Ginkgo to do exactly that. This novel strain has the ability to consume methionine in the human gut, helping to avoid excessive accumulation and conversion to homocysteine in plasma. High-throughput testing of codebase libraries by Ginkgo enabled SYNB1353 to advance from preclinical proof-of-concept to candidate strain in the space of a year.

“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.”

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. Researchers from Synlogic and Ginkgo will present preclinical data supporting advancement of SYNB1353 into IND-enabling studies at the International Congress of Inborn Errors of Metabolism 2021. The poster, Development of an Investigational Methionine-consuming Synthetic Biotic Medicine (SYNB1353) for the Treatment of Homocystinuria, will be available at the International Congress of Inborn Errors of Metabolism 2021 on November 21-23, 2021 in Sydney, Australia.

We’re so honored to be able to support Synlogic in building their incredible platform to provide potential new treatment options for people living with difficult to treat diseases. 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.

Read the full press release here.

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