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 Announces First Dosing of RT001 in patients with Infantile Neuroaxonal Dystrophy (INAD)

The U.S. Food and Drug Administration (FDA) has granted the company approval to conduct this trial as an open-label Phase 2/3 clinical trial in 15-20 patients. Due to the paucity of publicly available natural history of the progression of the disease,  Retrotope is currently also enrolling a prospective natural history study in a matched group of INAD patients who are not participating in the trial. (December 13, 2018)


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)

Parents of Leeds toddler with rare condition thank public as USA drug trial continues

Zoe was one of only 15 children worldwide picked for the clinical trial, and since launching a fundraising effort, parents Christine Hamshere and Steven Lightfoot have seen people donate more than £22,000 towards family trips to the USA to have treatment by Retrotope’s drug in New Jersey. Read more by clicking here.


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