Homocystine

Aeglea BioTherapeutics Submits CTA Application for its Novel Engineered Human Enzyme Designed to Treat Homocystinuria (ACN00177)

Retrieved on: 
Monday, January 13, 2020

Currently, people diagnosed with homocystinuria face a debilitating chronic disease, poor quality of life and inadequate treatments, including severe lifelong dietary restrictions, said Anthony G. Quinn, M.B.

Key Points: 
  • Currently, people diagnosed with homocystinuria face a debilitating chronic disease, poor quality of life and inadequate treatments, including severe lifelong dietary restrictions, said Anthony G. Quinn, M.B.
  • "Aegleas innovative platform has engineered a novel enzyme which, by reducing plasma homocysteine levels, has the potential to transform the patient experience with this challenging disease.
  • Aeglea is developing ACN00177 for the treatment of patients with cystathionine beta synthase (CBS) deficiency, also known as Classical Homocystinuria.
  • ACN00177 has been designed as a novel recombinant human enzyme, which degrades the amino acid homocysteine and its related homocystine dimer.