Targeting Repeat Expansion Disorders with Next-Gen Antisense Drugs


Manage episode 229681972 series 60790
By Discovered by Player FM and our community — copyright is owned by the publisher, not Player FM, and audio streamed directly from their servers.
A number of rare, genetic diseases including Huntington’s disease and myotonic dystrophy belong to a group of conditions known as repeat expansion disorders. Though it's normal to have pieces of repeating genetic code, these diseases involve an abnormally high number of repeats that result in the production of toxic proteins. NeuBase Therapeutics is using its platform technology to produce antisense drugs that it believes have distinct advantages over existing antisense drugs today. NeuBase recently announced a reverse merger with Ohr Pharmaceutical that propels the pre-clinical stage company onto the Nasdaq market. We spoke to Dietrich Stephan, CEO of Neubase, about repeat expansion disorders, the company’s platform technologies, and why he believes it has distinct advantages over existing therapies that target RNA.

240 episodes available. A new episode about every 0 hours averaging 23 mins duration .