Development of an Oral Methionine-Gamma-Lyase Enzyme for the Treatment of Homocystinuria

- 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. Project Number: 1R44HD118842-01A1 | Fiscal Year: 2025 | NIH Institute/Center: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Principal Investigator: Vasu Sethuraman | Institution: SYNTIS BIO INC, Boston, MA | Award Amount: $347,016 | Activity Code: R44 | Study Section: Special Emphasis Panel[ZHD1 DSR-A (10)] View on NIH RePORTER: https://reporter.nih.gov/project-details/11253870