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 today announced that it has supplied its drug RT001 for the initiation of an expanded access (EA) trial to treat patients with amyotrophic lateral sclerosis (ALS). This EA program in ALS, requested by renowned investigators at major medical centers, is expected to enroll a few patients at each site and is intended to provide information to guide the design of future randomized placebo-controlled trials.

(September 17, 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)