RARE Daily

DTx Pharma Receives Grant to Advance RNA-based Therapeutics for CMT

September 22, 2020

Rare Daily Staff

DTx Pharma said it has received a new funding grant from the National Institutes of Health’s National Institute of Neurological Disorders and Stroke to advance its research program for Charcot-Marie Tooth type 1A, a genetically driven, rare neuromuscular disease. 

Charcot-Marie-Tooth (CMT) disease is a group of rare inherited disorders that affect the peripheral nerves that run from outside the brain and spine. Defects in at least 30 genes cause different forms of this disease. Common symptoms may include foot drop, foot deformity, loss of lower leg muscle, numbness in the foot or leg, feet hitting the floor hard when walking, and weakness of the hips, legs, or feet. There is currently no cure for Charcot-Marie-Tooth disease, but physical therapy, occupational therapy, braces and other orthopedic devices, pain medication, and orthopedic surgery can help manage and improve symptoms.

DTx is advancing a preclinical program using its proprietary platform technology called FALCON (Fatty Acid Long Chain OligoNucleotide), to suppress the overexpression of PMP22, the protein that causes CMT type 1A.

The preliminary data supporting the funding came in large part due to seed funding from the Charcot Marie Tooth Research Foundation. To date, DTx has received more than $2 million in funding grants to support research and advance their pipeline of novel RNA-based therapeutics.

“We invested in DTx’s technology because of its potential to be groundbreaking for CMT, and in a very short period, DTx has generated significant success,” said Susan Ruediger, CEO of the CMT Research Foundation. “This partnership exemplifies how a relatively small investment from the CMT Research Foundation can lead to a significant follow-on capital for a promising project to develop treatments for CMT. This wouldn’t have happened without the CMTRF’s early investment.”

DTx Pharma’s proprietary delivery technology platform, FALCON utilizes fatty acids as targeting ligands to enable the delivery of oligonucleotide therapies to tissues and cell types throughout the body. In preclinical studies, DTx has demonstrated cellular uptake and broad activity of oligonucleotides in the retina, muscle, heart, neurons, T cells and many other specialized cell types.

“With this non-dilutive funding and support from our partners, we are well-positioned to develop a clinical candidate for CMT1A that is effective at suppressing the disease-driving gene, PMP22,” said Arthur Suckow, CEO of DTx Pharma.

Photo: Susan Ruediger, CEO of the CMT Research Foundation

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