Activity of Synb1353, an Investigational Methionine-Consuming Synthetic Biotic Medicine, in an Acute Nonhuman Primate Model of Homocystinuria

Perreault, Mylène; Gerson, Erik; Means, Jillian; James, Michael J.; Jessel, Michael J.; Moore, T. C.; Reeves, Analise Z.; Lubkowicz, David; Hava, David L.

doi:10.1016/j.ymgme.2022.01.075

Cited by 2 publications

(1 citation statement)

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“…It follows the same concept as previously reported for the synthetic live bacterial therapeutic agent, SYNB1618, for phenylketonuria, an inborn error of phenylalanine metabolism (Isabella et al, 2018; Puurunen et al, 2021). The development and the first preclinical data for SYNB1353 were presented at the 14th International Congress of Inborn Errors of Metabolism (Perreault et al, 2021). The genetic engineering of SYNB1353 involved both the modification of Met catabolism and Met uptake.…”

Section: Emerging Therapeutic Approaches To Hcumentioning

confidence: 99%

Recent therapeutic approaches to cystathionine beta‐synthase‐deficient homocystinuria

Majtán

Kožich

Kruger

2022

British J Pharmacology

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Cystathionine beta-synthase (CBS)-deficient homocystinuria (HCU) is the most common inborn error of sulfur amino acid metabolism. The pyridoxine non-responsive form of the disease manifests itself by massively increasing plasma and tissue concentrations of homocysteine, a toxic intermediate of methionine metabolism that is thought to be the major cause of clinical complications including skeletal deformities, connective tissue defects, thromboembolism and cognitive impairment. The current standard of care involves significant dietary interventions that, despite being effective, often adversely affect quality of life of HCU patients, leading to poor adherence to therapy and inadequate biochemical control with clinical complications. In recent years, the unmet need for better therapeutic options has resulted in development of novel enzyme and gene therapies and exploration of pharmacological approaches to rescue CBS folding defects caused by missense pathogenic mutations. Here, we review scientific evidence and current state of affairs in development of recent approaches to treat HCU.

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Section: Emerging Therapeutic Approaches To Hcumentioning

confidence: 99%