Retrotope is at the forefront of developing innovative therapies aimed at combating degenerative diseases, particularly those linked to oxidative stress and lipid peroxidation (LPO). The company utilizes a proprietary drug discovery platform to create isotopically stabilized synthetic versions of polyunsaturated fatty acids (PUFAs), which are crucial for maintaining cellular integrity. Their lead candidate, RT001, is a synthetic linoleic acid designed to down-regulate LPO, thereby protecting cells from degeneration. This compound is currently being investigated for its efficacy in treating several orphan neurodegenerative diseases, including infantile neuroaxonal dystrophy (INAD), Friedreich's ataxia, amyotrophic lateral sclerosis (ALS), and progressive supranuclear palsy (PSP).

In addition to RT001, Retrotope is advancing RT011, an isotopically stabilized synthetic docosahexaenoic acid (DHA), aimed at addressing dry age-related macular degeneration (AMD). Preclinical studies have shown that RT011 provides dose-dependent protection against retinal damage caused by oxidative stress, preserving visual function in animal models. This innovative approach not only targets the underlying mechanisms of these degenerative diseases but also offers a promising oral administration route, which could enhance patient compliance and treatment outcomes. Retrotope's commitment to developing first-in-class therapies highlights its potential to significantly impact the treatment landscape for these challenging conditions.

United States

Retrotope is developing a new class of drugs designed to combat the disease and cellular degeneration that results from lipid peroxidation (LPO). Their lead drug candidate, RT001, is currently in late-stage clinical trials for the treatment of several orphan neurodegenerative diseases with few or no treatment options. This include amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease), Friedreich’s ataxia (FA) and infantile neuroaxonal dystrophy (INAD).

Retrotope

Company Details