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), a high value untreated fatal orphan disease. Retrotope’s transformational platform technology of stabilized lipid drugs has the potential to treat a wide variety of other mitochondrial myopathies, but also show promise for diseases such as Parkinson’s, Alzheimer’s, diabetic retinopathy, and others.

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 announces Phase I/II Clinical Trial Results of RT001 in Treatment of Friedreich’s ataxia. First-in-human trial achieves safety, tolerability, and PK goals, with early signals of efficacy .

(September 15, 2016)

Presentation by Dr. Harry Saal about how RT001 addresses the orphan disease Friedreich's ataxia.

(October 12, 2015)