Inborn errors in metabolism (IEM) result in diminished function of biochemical pathways that lead to severe, chronic medical conditions with few or no treatment options. Current therapies are restricted to management of diet, limiting physical exertion, and hospitalization in times of metabolic crisis due to infection, fever, or other physiologic stressors. EnergXT offers new alternatives for treating fatty acids oxidation (FAO) and energy metabolism disorders through a personalized medicine approach. The individualized testing platform and therapeutic solutions provided by EnergXT address the wide variability in disease presentation with a custom treatment plan for each patient.

Technology Description

Using our molecular and cellular testing platform, we have shown that treatment with a reformulation of trimetazidine (TMZ) — an ischemic heart disease generic drug approved outside the US — significantly improves activity of defective mitochondrial fatty acid oxidation enzymes in cells from patients with FAO and metabolic disorders. TMZ causes FAO intermediates to act as chaperones, stabilizing defective enzymes. In combination with other metabolic treatments, our solutions will increase the function of the fatty acid oxidation pathway to dramatically improve energy metabolism. EnergXT’s ultimate goal is to improve the quality of life for infants, children, and adults with fatty acid oxidation disorders, and to eliminate morbidity and mortality caused by these devastating diseases.

Advantages

  • Comprehensive therapeutic solutions to treat patients with fatty acids oxidation disorders
  • Custom molecular diagnostic and cellular platform can assess efficacy and customize multi-drug prescription and adjustment for intelligent, personalized therapy
  • Eliminates the threat of decompensation
  • Improves patients’ quality of life and enables them to exercise freely

Applications

Stage of Development

  • Clinical trials and preclinical testing of diagnostics and dosage
  • Developing and testing combination therapy in vitro
  • Ongoing animal studies

IP Portfolio