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.


Mikhail Shchepinov, Chief Scientific Officer at Retrotope, Inc., presenting at Undoing Aging 2019

Screen Shot 2019-08-16 at 13.01.41.png

Retrotope Announces Full Enrollment of Phase 2/3 trial in patients with Infantile Neuroaxonal Dystrophy (INAD)

Retrotope announced today that it has fully enrolled all patients in the first clinical trial of a possible treatment for infants with the ultra-rare neurodegenerative disease, 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. With FDA guidance Retrotope is currently also enrolling a prospective natural history study using the same evaluation metrics in a matched group of INAD patients who are not participating in the trial. RT001, the test drug, is the first-in-class of a new category of drugs called D- PUFAs (deuterated polyunsaturated fatty acids), which are designed to protect against free radical damage to omega 3 and 6 fats that results in cell death and is a hallmark of numerous neurodegenerative diseases including INAD.  (August 7, 2019)


Retrotope Completes Series C Financing

Retrotope announced today that it closed a Series C financing, enabling expansion of its pipeline of novel first-in-class drugs called D-PUFAs (deuterated polyunsaturated fatty acids). Round co-led by existing inside investors and new outside family office. The $20MM round was oversubscribed. Mehta Family Partners, an outside family fund, co-led the round with Timur Artemev, a UK-based inside investor (along with other insiders who re-invested). Also joining the round was Retrotope’s first institutional investor, Morningside Venture Investments, Ltd.(July 19, 2019)

Publication in the "Frontiers in Psychology" journal entitled “Deuterated Polyunsaturated Fatty Acids Reduce Oxidative Stress and Extend the Lifespan of C. elegans”.

(May , 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.


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