Armatus Bio Inc., Columbus, Ohio
A Potential “Precision Drug Therapy” For 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. 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.