CMT Research Foundation Partner DTx Pharma Wins Grant from National Institutes of Health for CMT1A Gene Therapy Program

Sep 22, 2020 | CMTRF Funded Research, Drug Development, Research news

CMT Research Foundation partner DTx Pharma, Inc., has won a grant from the National Institutes of Health (NIH) to further fund its work on CMT. The investment is significant because the CMT Research Foundation gave DTx its first CMT-related grant just nine months ago. This partnership exemplifies how our initial research investments, made possible one dollar at a time by generous donors, can lead to significant follow-on capital and promising projects to develop CMT treatments.  The CMT Research Foundation first approached DTx to consider CMT when we learned about their technology. Our grant initiated a CMT program at DTx, and in just a few months, DTx has generated significant success. DTx’s interest in CMT and the subsequent follow-on funding wouldn’t have happened without the this early investment.

“The CMT Research Foundation and the NIH have supported groundbreaking research for rare diseases like CMT. We are pleased to continue to partner with organizations that share our commitment to developing therapies where none exist. 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.”

Arthur T. Suckow, CEO of 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 to suppress the overexpression of PMP22, the protein that causes CMT1A.

Read more about all the research projects and partnerships the CMT Research Foundation is funding.

Be Part of the Next Big Breakthrough for CMT

While no treatments or cures currently exist for CMT, the science to change that does.

Your donation to the CMT Research Foundation will fund cutting-edge research that’s solely focused on drug development and advance more innovative partnerships like our current collaboration with DTx Pharma. Together, we can change the course of CMT during our lifetime — and for every future generation.