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DTx Pharma Receives New Funding Grant to Advance RNA-based Therapeutic Programs for Charcot-Marie Tooth (CMT)

SAN DIEGO, Sept. 22, 2020 /PRNewswire/ -- DTx Pharma, Inc. (DTx), a privately-held biotechnology company creating novel RNA-based therapeutics to treat the genetic drivers of disease, announced today that the company has received a new funding grant to advance its research program for Charcot-Marie Tooth (CMT), a genetically-driven, rare neuromuscular disease.  DTx is advancing a preclinical program using its proprietary platform technology, FALCON™ (Fatty Acid Long Chain OligoNucleotide), to suppress the overexpression of PMP22, the protein that causes CMT1A.

The new grant announced today comes from the NINDS at the National Institutes of Health (NIH).  The preliminary data supporting the funding of this application came in large part due to seed funding and resulting preliminary data from the Charcot Marie Tooth Research Foundation—the world's leading organization advancing research for CMT. To date, DTx has received over $2M in funding grants to support research and advance their pipeline of novel, RNA-based therapeutics. 

"The CMT Research Foundation and the NIH have supported ground-breaking research for rare diseases like CMT and we are pleased to continue to partner with organizations that share our commitment to developing therapies where none exist," said Arthur T. Suckow, CEO of DTx Pharma.  "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."

"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," says Susan Ruediger, CMTRF's CEO.  "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."

About DTx Pharma

DTx Pharma, Inc. is a privately-held biotechnology company creating novel RNA-based therapeutics to treat the genetic drivers of disease.  The company's proprietary delivery technology platform, FALCON (Fatty Acid Long Chain OligoNucleotide), 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. Based upon the novelty, IP position and data demonstrating the effectiveness of its proprietary fatty acid motifs, DTx has previously received investment, nondilutive funding and other forms of support from Eli Lilly, Eye CRO and the CMT Research Foundation (CMTRF) for its ocular and muscle programs and has received multiple grant awards from the NIH (NCATS, NIA), and the TPEP program of Alzheimer's Association and Rainwater Charitable Trust to advance its efforts to treat CNS diseases.  To learn more about DTx Pharma, please visit and follow DTx on Twitter @DTxPharma.

About CMT Research Foundation

The CMT Research Foundation (CMTRF) is focused solely on delivering treatments and cures for CMT. Founded by two patients who are driven to expedite drug delivery to people who live with CMT globally, the organization funds research for drug development. The 501(c)(3) federal tax-exempt organization is supported by personal and corporate financial gifts.


Amy Conrad
Juniper Point

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Last Updated: 22-Sep-2020