GLENOLDEN, PA — The Charcot-Marie-Tooth Association (CMTA) has announced its participation in the Seed Series Extension syndicate for Armatus Bio, Inc., a rising biotechnology firm based in Columbus, Ohio. The investment amount remains undisclosed.
Armatus Bio is developing a unique gene therapy clinical candidate aimed at targeting CMT type 1A, the most prevalent form of Charcot-Marie-Tooth disease (CMT). An inherited peripheral neuropathy, CMT principally affects the long nerves in hands and feet, impacting around 3 million people worldwide. Despite its prevalence, CMT often faces misdiagnosis or late diagnosis, with no FDA-approved therapies currently available for any type of CMT.
CMT1A results from a duplication of the PMP22 gene, leading to overexpression of the PMP22 protein, which in turn destabilizes and degrades the myelin sheath surrounding nerves. This results in increasing nerve loss over time and disease progression. Armatus Bio’s treatment, ARM-101, is an adeno-associated virus (AAV) based gene therapy that encodes a small interfering RNA (siRNA). This siRNA works by reducing the production of PMP22 protein in Schwann cells.
Armatus Bio has already demonstrated the efficacy of this approach in CMT1A mouse models. The company is currently conducting studies that will enable them to submit an Investigational New Drug (IND) application to the FDA in 2025. If cleared, this will result in a Phase 1 clinical trial to evaluate the safety, tolerability and potential clinical benefit of ARM-101 in patients with CMT1A.
Armatus CEO Rachel Salzman, DVM, expressed her gratitude to the CMTA for its support, stating, “Our ARM-101 development studies are particularly important for the CMT community because the data generated are designed to answer critical questions about how we can effectively deliver genetic medicines to the right parts of the body to meaningfully address this disease.”
Thomas Dubensky, Ph.D., a CMTA board member, stated that Armatus is the CMTA’s first investment from its new venture philanthropy arm. This venture will make selected early-stage investments in biotech companies developing unique and promising therapies against all types of CMT. This strategic move marks a significant step in the journey towards developing effective treatments for this prevalent disease.
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