Multiple Sclerosis (MS) is an autoimmune disease that damages the myelin coating around nerve fibres, causing disruption of nerve signals and a variety of symptoms of variable severity. Myelin is the sleeve of fatty tissue that protects and insulates nerve fibres, like insulation around a wire. Myelin helps electrical signals travel along nerve fibres. Damage to myelin and nerve fibres is called demyelination. The scar tissue that results is called sclerosis.
NeuOrphan has identified what we believe is a root cause of progressive Multiple Sclerosis—a deficiency in the brain’s thyroid hormone transporter, MCT8, which disrupts myelin repair by causing the death of myelin-producing cells (oligodendrocytes).
Our lead candidate, Dioprotectome™, is a small molecule that restores remyelination by bypassing the MCT8 pathway and protecting oligodendrocytes from immune damage. It has shown compelling results in both animal models and human cell studies, reversing neurological decline and promoting regeneration of damaged nerve fibres.
Unlike current MS treatments, which only suppress immune symptoms, Dioprotectome™ offers the potential to halt—and even reverse—the course of MS.
Investment highlights
Blockbuster drug potential driving potential for unicorn valuation
Scientific program significantly derisked after 15 years of research
Multiple exit opportunities
Huge social impact
Corporate website: https://neuorphan.com/
Problem and Solution
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