The CMT Research Foundation has invested in a project at Armatus Bio, Inc. that will advance a novel miRNA gene therapy candidate for CMT type 1A. Principal Investigator Dr. Brian Price and his colleagues aim to harness the potential of microRNAs (miRNAs), snippets of genetic code that have the ability to enter the cells of the body and, with precision, modify patterns of gene activity.

In the peripheral nerve, cells called Schwann cells provide insulation around nerve fibers to support normal nerve function. Schwann cells contain a gene named PMP22. This job of PMP22 is making just the right amount of myelin to build and maintain the nerve’s insulating sheath. Unfortunately, in people with CMT1A, PMP22 is overactive. As a result, too much myelin is expressed in Schwann cells, and this disrupts normal nerve function and is a root cause of the development of the symptoms and debilitating impact of CMT1A. If PMP22 function could be normalized – to stop myelin overproduction – this could potentially allay or reverse the pathology of CMT1A.

In this new CMTRF-funded study the clinical candidate, called ARM-101, will deliver the miRNA inside a viral carrier called an adeno-associated virus (AAV). In animal-based studies, ARM-101 will be delivered by a single injection adjacent to the spinal cord, in a standard clinical manner that is used to deliver many other drugs including painkillers. The goals of this study include understanding if and how ARM-101 is reaching the Schwann cells and having the desired effect; learning more about the drug’s dynamics while in the body, dosage versus outcome; and ultimately, achieving clinically meaningful functional improvements.

“This study is particularly important for the CMT community because it will provide important answers to critical questions about how we can effectively deliver genetic medicine to the right parts of the body to address the effects of this disease,” said Dr. Price. “By gathering evidence that the medicine directly affects the Schwann cells and can may normalize PMP22 before we move into human trials, we can demonstrate that this has real potential to provide benefit to the thousands of people living with CMT1A today in the U.S. and around the world.”

If the results of this study and those of other ongoing research are successful, and following discussions with U.S. regulatory authorities, Armatus Bio plans to advance toward clinical trials in humans.

ARM-101 continues a candidate therapeutic’s journey for CMTRF. The core technology of the ARM-101 was first developed through a previous CMTRF research award to Dr. Kleopas Kleopa from the Cyprus Institute of Neurology and Genetics and Dr. Scott Harper from National Children’s Hospital in Ohio.

Armatus Bio subsequently licensed this technology, and is now advancing it toward the clinic with CMTRF support. CMTRF is proud to have funded the early foundational studies for the development of ARM-101, and to now be providing a bridge of support to take those early-stage discoveries closer to a treatment for CMT1A