Helixmith has published a study that confirmed the efficacy of AV6-H’s, a gene therapy using adeno-associated virus (AAV) vectors, in treating neuromuscular disease.

The company is building a portfolio for neuromuscular disorders, and AV6-H is the third candidate that the company has released following VM202 and AV1-H.

Helixmith developed AV6-H by inserting the hepatocyte growth factor gene after excising the AAV subspecies to deliver the therapeutic substance with a simple intramuscular injection efficiently.

Its researchers confirmed the treatment’s various therapeutic effects such as peripheral nerve regeneration, improvement of nerve function, an increase of muscle mass, and improvement of motor function in a preclinical “nerve crush” model, the company said.

Also, in a SOD1-G93A transgenic mice model, a model commonly used for Lou Gehrig’s disease, they confirmed that AV6-H injection had the effects of improving muscle mass, alleviating symptoms, and mortality.

“The main implication of this study is we have managed to develop a treatment for patients with various types of neuromuscular disorders that can significantly improve the quality of life with simple intramuscular injections,” the company said in a release.

Helixmith plans to conduct a variety of studies and clinical trials with AV6-H for use in unidentified neuromuscular disorders and prepare to conduct U.S. phase 1 clinical trial for one or two of the indications by 2021 to 2022.

“There are dozens of neuromuscular disorders with most of the causes still unknown,” Helixmith CEO Kim Sun-young said. “Intramuscular injections can increase the quality of life of patients suffering from degenerative nervous system muscle diseases in a relatively safe and simple manner.”