CMT2S Breakthrough

In a recent announcement, Vanda Pharmaceuticals Inc. shared that the U.S. Food and Drug Administration (FDA) has approved the Investigational New Drug (IND) application for its VCA-894A compound. This approval marks a significant step forward for community members who have CMT2S. CMT2S is a very rare and often debilitating autosomal recessive type of CMT.

A potential treatment for CMT2S, Vanda’s VCA-894A compound is a novel (new) antisense oligonucleotide (ASO) that targets certain variants (mutations) within the IGHMBP2 gene, which is implicated in CMT2S. ASOs have shown promise in treating various neurodegenerative and neuromuscular disorders. Although this is not a project funded by the CMTA, we have several ongoing ASO projects for other types of CMT.

“We at Vanda are inspired by the privilege and opportunity to work towards providing a personalized treatment to an individual with Charcot-Marie-Tooth. The pioneering work of Dr. Smieszek creates a roadmap for bringing gene specific information into the design of novel therapeutics.“ said Dr. Mihael Polymeropoulos, CEO of Vanda Pharmaceuticals.” Sanda Smieszek, Ph.D., is Vanda’s head of genetics whom we recognized in our recent “40 under 40” initiative in which we featured 40 up-and-coming CMT researchers who are under 40 years old. Dr. Smieszek is Vanda’s lead investigator for its VCA-894A program.

How Can I Participate?

To participate in this research, click the button below to register with the CMTA’s Patients as Partners in Research today! Registering ensures you are the first in line for any CMT research opportunity you might be eligible for and are interested in, including this one when Vanda is ready to open its trial for CMT2S. Registering will also ensure you are kept up to date on all the latest news and developments in CMT research. Registration is free and all who have CMT are invited to join.

Register Today

Published: February 15, 2024