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.
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.
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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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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