Our initial targets are orphan diseases, such as ALS, which benefit from an accelerated FDA approval process and market exclusivity
Amyotrophic Lateral Sclerosis (ALS, or Lou Gehrig’s Disease)
Amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease) is a neurodegenerative disease of the motor neurons in the brain and spinal cord that control muscle movement. The degeneration of motor neurons in ALS causes muscle spasticity, progressive weakness and difficulty with speaking, swallowing and breathing. ALS is the most common of the five known motor neuron diseases, affecting approximately 30,000 people in the U.S. The only approved treatment for ALS has minimal impact on patient survival.
Transverse myelitis is a rare disease caused by inflammation of one level of the spinal cord. Attacks of inflammation in transverse myelitis damage or destroy the myelin sheath around nerve cell fibers, likely by damaging the oligodendrocytes that produce myelin. An estimated 44,000 people in the U.S. are affected by transverse myelitis, for which no effective cure currently exists.
Multiple sclerosis (MS) is a currently incurable inflammatory disease of the central nervous systems that leads to the damage or destruction of myelin in the brain and spinal cord. While the cause of MS is not clear, the underlying mechanism of the disease is thought to be either autoimmune destruction or oligodendrocyte failure. In the U.S., approximately 400,000 people are living with MS and more than 10,000 new patients are diagnosed each year.
Spinal Cord Injury
Spinal cord injury is a broad term covering damage to any part of the spinal cord or the nerves at the end of the spinal canal. Spinal cord injuries often results in permanent alterations in strength, sensation and other body functions below the level of the injury. An estimated 12,000 people sustain severe spinal cord injuries each year, and there is currently no way to reverse the damage done to the spinal cord.