Reinforcing the Molecules of Life to Combat Degenerative Diseases
Human cells are continuously damaged by oxidative stress that is driven by lipid peroxidation (LPO). The resulting cellular degeneration contributes to a wide array of devasting degenerative diseases.

Retrotope is developing a pipeline of proprietary therapeutics designed to treat degenerative disease by down-regulating LPO in order to protect against cellular degeneration.

At Retrotope, we are pioneering the development of first-in-class therapies for devastating degenerative diseases - ranging from orphan neurodegenerative indications to large market degenerative conditions - for which there are few to no treatment options.

We are leveraging our proprietary drug discovery platform technology, along with our dedicated team of scientific leaders and industry veterans, to create novel, disease-modifying therapies designed to combat the oxidative stress and cellular degeneration that arise from lipid peroxidation.

Compound Indication Discovery/Preclinical Phase I Phase II Phase III
Stabilized Linoleic Acid (LA,
ethyl ester)

Infantile neuroaxonal dystrophy (INAD)

Friedreich's ataxia (FA)

Amyotrophic lateral sclerosis (ALS)

Progressive supranuclear palsy (PSP)

Stabilized Docosahexaenoic
acid (DHA, ethyl ester)
Dry Age-Related Macular Degeneration (Dry AMD) and geographic atrophy (GA)
Other stabilized PUFAs Huntington’s disease (HD), Spinocerebellar ataxia (SCA) and Multiple system atropy (MSA)
Positive Results from Studies of Retrotope’s RT011 in Animal Models of Retinal Degeneration to be Featured in Oral Presentations at 2nd Annual Dry AMD Therapeutic Development Summit
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Retrotope Reports Data from Phase 2/3 Clinical Trial of RT001 and Concurrent Natural History Study in Patients with Infantile Neuroaxonal Dystrophy (INAD)
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Retrotope Announces Completion of Enrollment in Phase 2 Study of RT001 in Patients with Progressive Supranuclear Palsy (PSP)
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First-in-Class, Oral Drug Candidate to be Evaluated in Randomized, Double-Blind, Placebo-Controlled Study in Patients with Debilitating Neurodegenerative Disease
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The study’s enrollment target of 40 patients was exceeded in less than six weeks, highlighting the high demand for participation in the trial featuring a novel treatment approach for ALS. Based on this rapid patient enrollment, Retrotope expects...
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The first patient was dosed on March 10, with an additional eight patients enrolled during the study’s first week. Clinical investigators have already reported additional patient interest beyond the 40 subjects planned for the trial. As such,...
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