Federal grant · project grant (b)
Development of an Oral Methionine-gamma-lyase Enzyme for the Treatment of Homocystinuria - Abstract - Development of an Oral Methionine-gamma-lyase Enzyme for the Treatment of Homocystinuria Syntis Bio Is Developing SYN-202, a Novel Methionine Gamma-lyase (MGL) Enzyme Therapy Aimed at Correcting the Metabolic Dysregulation in Homocystinuria (hcu), a Rare Pediatric Disorder Caused by Mutations in the Cystathionine-β-synthase (CBS) Enzyme. This Genetic Defect Leads to the Toxic Accumulation of Methionine and Homocysteine, Disrupting Critical Metabolic Pathways and Resulting in Severe Neurological Symptoms, Developmental Impairments, and Life-threatening Metabolic Crises. Current Treatments, Which Primarily Rely on Dietary Methionine Restriction, Are Challenging for Young Children and Patients in Resource-limited Settings to Adhere to, Leading to Poor Compliance and Poor Outcomes. Syn- 202 Offers a More Effective and Patient-friendly Alternative by Enzymatically Replicating the Effects of Dietary Methionine Restriction in a Convenient Oral Formulation. Additionally, Syn- 202 Is Engineered for Proteolytic and PH Stability, Overcoming the Delivery Challenges That Often Limit the Efficacy of Oral Biologics, Ensuring Consistent and Reliable Activity.
Committed
$347,016
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