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) and Infantile Neuroaxonal Dystrophy (INAD), both untreated fatal orphan diseases. 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.

For a short two page overview of the status and opportunity created by Retrotope, please click here.


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 RT001 Clinical Data Presentation at the 2019 American Academy of Neurology (AAN) Annual Meeting

Retrotope announced positive findings from an expanded access study of the company’s lead candidate, RT001, in a patient with LOTS, as presented at the 71st AAN Annual Meeting, being held in Philadelphia, PA, May 4-10, 2019.  The study showed that administration of RT001 to a 44-year-old LOTS patient appears to have generated improvements in a variety of patient reported outcomes (choking coughs, strength, falls) and in objective measures of performance (timed walks, quantified speech measures, Archimedes spiral). Effects were measured in daily or weekly diaries and test sessions at home.  (May 7, 2019)


Retrotope today received a notification from the Committee for Orphan Medical Products of the European Medicines Agency (EMA) that its request for orphan medicinal product designation for the treatment of INAD with RT001, a chemically-fortified polyunsaturated fatty acid drug, has been granted by the European Commission.  Retrotope is currently enrolling a Phase 2/3 trial in the US for INAD, an ultra-rare, severe childhood neurodegeneration due to an inborn error in PLA2G6. (March 13, 2019)

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