Novartis CMT1A Update

by | Oct 23, 2023 | 0 comments

CMT Community Statement October 2023

Dear Charcot-Marie-Tooth disease (CMT) Community,

In July 2023, Novartis announced the acquisition of DTx Pharma, a San-Diego based, preclinical stage biotechnology company focused on leveraging its proprietary FALCON platform to develop siRNA therapies for neuroscience indications.

DTx’s lead investigational program, DTx-1252 targets the cause of CMT1A, the overexpression of the protein PMP22.  This overexpression causes the myelin sheath that supports and insulates nerves in the peripheral nervous system to function abnormally. DTx-1252 decreases the expression of this protein in Schwann cells.

Novartis is in the process of onboarding information from DTx and developing next steps. We look forward to continuing DTx’s therapeutic programs and bringing new hope to patients with neuromuscular and other neurological disorders for which there have historically been few treatment options, including CMT1A.

Novartis is committed and excited to work with our partners in the CMT community to make a difference in the diagnosis, treatment, and care of those affected by CMT.  Novartis deeply appreciates the collaboration and support of the CMT community. Our CMT advocacy partners are strong allies who we look forward to working with to deepen our understanding of this disease as we advance promising science with transformative potential for CMT1A.

We look forward to collaborating closely with the CMT community and commit to providing you with updates on a regular basis as we have more information available to share.  We thank you for your engagement and will continue to keep you informed of our progress.


The Novartis CMT1A Development Team

© 2023 Novartis Gene Therapies, Inc.

Bannockburn, IL 60015

US-UNB-23-0065 10/2023

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