Helixmith said that it has administered the first dose of VM202 to patients with Charcot-Marie-Tooth (CMT) disease, as part of its local phase 1 and 2a clinical trial.

During the trial, a research team, led by Professor Choi Byung-ok at Samsung Medical Center, plans to evaluate the safety and tolerability of VM202 in 12 CMT patients.

Helixmith plans to secure safety and efficacy data through this clinical trial. If the company manages to obtain positive results through the trial, it intends to conduct additional global clinical trials in the U.S. and Europe.

"CMT is a disease in which the muscles of the arms and legs atrophy and gait disorders occur due to the motor and sensory nerves' progressive damage. It is one of the ‘most common rare diseases,’” the company said. "Notably, CMT type 1A is a CMT that occurs due to an abnormality in the PMP22 gene and accounts for 40 percent of the total CMT."

There are about 8,000 CMT1A patients in Korea, and more than 1.2 million patients worldwide.

"Despite such a large number of patients, there are still no drugs approved by the U.S. Food and Drug Administration," the company said.

Helixmith CEO Kim Sun-young said, "Since the U.S. FDA has already designated VM202 as regenerative medicine advanced therapy (RMAT) for diabetic neuropathy (DNP), the company expects the treatment will likely have a therapeutic effect on CMT treatment."