According to a story from GlobeNewswire, the biopharmaceutical company Ovid Therapeutics Inc. and Columbia University have recently entered a collaborative partnership that will be focused on developing gene-based treatments for KIF1A associated neurological disorder (KAND) and other rare diseases. As there are no current treatments developed specifically for this group of disorders, a disease-modifying therapeutic would be a major breakthrough. Ovid is focused on developing treatments for rare neurological disorders.
About KIFA Associated Neurological Disorder (KAND)
KIF1A associated neurological disorder (KAND) is a group of very rare genetic diseases that are linked to mutations and alterations affecting the KIF1A gene. This gene is responsible for the production of a certain protein that is only present in the neurons and brain; the function of the protein remains poorly understood. The symptoms of the disorder varies between patients, but a frequent manifestation is spastic paraplegia, or gait disorder, in which the leg muscles weaken and patients often lose their walking ability. Other symptoms may include loss of touch sensation in the hands and feet, developmental delays, changes to muscle tone, joint contractures, cerebellar atrophy, vision problems, peripheral neuropathy, coordination loss, facial paralysis, uncontrolled eye movements, seizures, scoliosis, and tremors. Treatment is based on how symptoms present and often requires collaboration between a team of specialists. More effective therapies are urgently needed for KIF1A associated neurological disorder. Around 150 diagnosed patients are known to science. To learn more about KIF1A associated neurological disorder, click here.
Finding Treatment Targets
In a disease as rare and poorly understood as KIF1A, the first goal of the research partnership will be identifying potential molecular targets for treatment development. The company will be collaborating closely with Dr. Wendy Chung, who is the leader of the Precision Medicine Resource and Chief of Clinical Genetics at the university’s Irving Medical Center. She is widely recognized as a leading advocate and researcher for rare diseases and was honored with the National Organization for Rare Disorder’s Rare Impact Award in 2019.
“My laboratory is committed to discovering innovative new targets and therapies to address rare neurological diseases with few or no treatment options.” – Dr. Wendy Chung.
This new research partnership is a new source of hope for the KIF1A associated neurological disorder rare disease community. Read more about this announcement here.
Check out this video about KIF1A patients below: