LPO and Degenerative Disease

All healthy human tissues undergo a physiological process of cellular degeneration and repair that arises naturally from lipid peroxidation (LPO). LPO occurs when inhaled oxygen is converted into reactive oxygen species (ROS), including free radicals, which in turn oxidize the polyunsaturated fatty acids (PUFAs) that make up cell and mitochondrial membranes throughout the body. PUFAs, which are vital to healthy cellular function, are particularly susceptible to LPO due to their inherent instability. When in balance, LPO is a process that initiates, propagates and terminates in a timely fashion, playing an important role in the natural cycle of cellular degeneration and repair.

However, it is well-established that a wide range of serious degenerative diseases are precipitated when the LPO process is not terminated quickly enough, allowing free radicals to set off an uncontrolled chain reaction that degrades thousands upon thousands of vital PUFAs. The result is broad oxidative damage to phospholipid membranes and ultimately cellular degeneration.

The Retrotope Solution

To combat the oxidative stress and cellular degeneration that arises from LPO, Retrotope has created a proprietary drug discovery platform technology that utilizes an elegant chemistry-based solution to create novel, disease-modifying drugs. These first-in-class new chemical entities (NCEs) are isotopically stabilized synthetic versions of PUFAs that become an integral part of all membranes throughout the body. By replacing inherently unstable natural PUFAs with these chemically stabilized versions, Retrotope is able to remove the fuel that drives uncontrolled, disease-causing LPO, without impacting the critical role that PUFAs play within the body.

To date, the company has leveraged its platform to discover RT001, a clinical-stage isotopically stabilized, synthetic linoleic acid (LA), and RT011, an isotopically stabilized, synthetic docosahexaenoic acid (DHA).


Our lead development candidate, RT001, is in late-stage clinical trials for a range of orphan neurodegenerative diseases including infantile neuroaxonal dystrophy (INAD), Friedreich’s ataxia (FA), amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease) and progressive supranuclear palsy (PSP).

We expect 2021 to be a potentially transformational year for Retrotope as we anticipate data from our Phase 2/3 trial in INAD, our pivotal Phase 2/3 study in FA and our Phase 2 trial in ALS by the end of 2021.

J. Thomas (Tom) Brenna, Ph.D.

Dr. Brenna is professor of pediatrics, professor of human nutrition, and professor of chemistry at the University of Texas at Austin.

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Lex Van der Ploeg, Ph.D.

Dr. Van der Ploeg is an experienced biotechnology industry executive, scientist and entrepreneur with a track record of contributing both to the development of novel therapeutics, as well as successful M&A transactions.

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Jan Vijg, Ph.D.

Dr. Vijg is professor and chair, department of genetics, as well as the Lola and Saul Kramer Chair in Molecular Genetics, at Albert Einstein College of Medicine.

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Charles Stacey, MBBS, MRCS, MBA

Dr. Stacey is president and chief executive officer of Cerecin, a clinical-stage biotechnology company developing a platform of ketogenic compounds to treat neurological diseases.

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Peter K. Kaiser, M.D.

Dr. Kaiser is the Chaney Family Endowed Chair for Ophthalmology Research and a professor of ophthalmology at the Cleveland Clinic Lerner College of Medicine. He graduated magna cum laude with Highest Honors from Harvard College and magna cum...

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Eleonora (Nora) Lad, M.D., Ph.D.

Dr. Lad is a clinician scientist and retinal ophthalmologist with the primary goal of developing novel strategies for early diagnosis and treatment of age-related macular degeneration (AMD).

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Jeffrey S. Heier, M.D.

Dr. Heier is the co-president and medical director, director of the vitreoretinal service, and director of retina research at Ophthalmic Consultants of Boston (OCB), one of the largest multi-specialty ophthalmology practices in the United States....

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Karl Csaky, M.D., Ph.D.

Dr. Csaky is a vitreoretinal disease specialist who current serves as the T. Boone Pickens director, managing director and chief medical officer at the Clinical Center of Innovation for AMD at the Retina Foundation of the Southwest.

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Carl D. Regillo, M.D.

Dr. Regillo is chief of the retina service at Wills Eye Hospital. He received his medical degree from Harvard Medical School, completed his internship in internal medicine at Harvard’s Brigham and Women’s Hospital in Boston, and performed his...

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Robyn Guymer, MBBS, Ph.D., FRANZCO, FAHMS

Dr. Guymer is one of the world’s leading experts on AMD and is well respected for her work on early prognostic markers, highlighted by being credited as one of the first individuals to define nascent geographic atrophy through OCT.

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Plasma and Red Blood Cell Membrane Accretion and Pharmacokinetics of RT001
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The infantile neuroaxonal dystrophy rating scale (INAD-RS).
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Alpha synuclein aggregation drives ferroptosis
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