Retrotope has created a new category of drug platform to preserve and restore mitochondrial health in degenerative diseases. The unique mechanism of Retrotope’s platform prevents cellular damage and recovers cellular function damaged by lipid peroxidation due to oxidative stress. Retrotope’s first product candidate, RT001, is being clinically tested in Friedreich’s ataxia (FA), an untreated fatal orphan disease. Other stabilized lipid drugs based on Retrotope’s transformational platform technology have the potential to treat a wide variety of other mitochondrial myopathies and neurodegenerative diseases.
Retrotope's drug platform, deuterium stabilized PUFAs, are unique in drug discovery. Retrotope’s stabilized fatty acid drugs prevent lipid peroxidation damage from propagating, rapidly stopping the toxic chain reaction at its source. Because the fatty acids in membranes turn over rapidly, the dietary substitution of stabilized fatty acids creates cells fortified against damage.
Retrotope announced today that the U.S. Food and Drug Administration (FDA) has granted the company approval to conduct an open-label Phase 2/3 clinical trial of its investigational drug RT001 to evaluate efficacy and safety in patients with infantile neuroaxonal dystrophy (INAD).
(June 28, 2018)
Publication in the "The FEBS Journal" of Huntington's model results, entitled "Deuterium‐reinforced linoleic acid lowers lipid peroxidation and mitigates cognitive impairment in the Q140 knock in mouse model of Huntington's disease."
(June 22, 2018)