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 reported new data at NORD Summit® that demonstrate arrest of disease progression with treatment of RT001 in patients with infantile neuroaxonal dystrophy (INAD). “INAD is a rare and devastating disease that strikes the very young and there are no current available treatments,” said Peter G. Milner, M.D., CMO of Retrotope. “The two patients enrolled in an expanded access program and presented here have been treated with RT001 for more than one year and six months, respectively. Both show disease arrest and some improvement in several components contained in the INAD rating scale. In the meantime, we are preparing to initiate a pivotal study of RT001 in INAD patients with the objective of confirming its efficacy and safety in this patient population.” (October 14, 2018)


Parkinson’s Institute and Retrotope announced today a collaboration on a new drug therapy to treat patients with Progressive SupraNuclear Palsy (PSP). The study is expected to help guide the design of future randomized placebo-controlled trials in PSP.

RT001 is the first-in-class of a new drug category called deuterated polyunsaturated fatty acids, which are a novel approach to protect against lipid peroxidation damage resulting in cell death. (October 4, 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)


Book Chapter: Retrotope - The Next Big Thing

With the permission of McGraw Hill, a pre-publication copy of Chapter 13 from "Get to Aha! Discover Your Corporate DNA to Dominate the Competition" by Andy Cunningham can be downloaded by clicking here