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 the start of two single patient, Compassionate Use trials of RT001 at major university medical centers in Late Onset Tay Sachs (LOTS) disease and Familial Encephalopathy with Neuroserpin Inclusion Bodies (FEIN or neuroserpinosis).

(June 11, 2018)

Publication in the "Movement Disorders" Journal of first-in-human clinical trial results,  entitled "Randomized, clinical trial of RT001: Early signals of efficacy in Friedreich's ataxia."

(April 6, 2018)