Therapeutic Focus

A focus on inborn errors of metabolism

Organic acidemias are a collection of conditions known as inborn errors of metabolism. In these cases, an inherited genetic defect creates a missing essential enzyme that plays a key role in metabolism – the series of chemical reactions whereby the body derives energy and synthesizes the molecules it needs to function.

The body tries to counter these defects in intermediate metabolism by using alternate metabolic pathways. Nevertheless, problems arise either through a lack of energy, a lack of required metabolic products, or metabolites that build up and damage organs.

The collection of rare diseases characterized as organic acidemias affects one in 5,000 live births, or about the same as Type 1 diabetes. Many are diagnosed in utero or shortly after birth.

And in many cases, these are devasting diseases: brain manifestations include developmental disorders, optic neuropathies leading to blindness, and strokes and seizures. OAs can lead to early kidney failure, heart attacks in young children, weak muscles, skin and hair ailments, and a damaged immune system that turns a normal childhood infection into a serious health crisis.

Restoring metabolism, restoring health

Comet, through its MetaSwitch™ Platform, is pioneering ways to switch cellular metabolism back to health, restoring the normal metabolic functions that are interrupted by the genetic mistakes that cause OAs. Our preclinical research shows that our proprietary compounds are able to address a variety of these metabolic deficits, correcting them and restoring healthy metabolism. We have demonstrated an ability to have a significant impact on the disease manifestations that are a part of living with an organic acidemia condition.

Going forward, Comet Therapeutics is committed to making a difference in the lives of patients living with OAs. We are also exploring other diseases that affect energy metabolism and neuro-metabolism.

Devastating impact

Organic acidemias are inborn errors of metabolism that lead to severe impacts on organs throughout the body in babies and young children.