Phase 1/2a Trial of Gene Therapy Engensis for CMT1A Begins in Korea

Phase 1/2a Trial of Gene Therapy Engensis for CMT1A Begins in Korea
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Helixmith has launched a Phase 1/2 clinical trial in South Korea to investigate its gene therapy product Engensis (VM202) for the treatment of muscle symptoms in people with Charcot-Marie-Tooth disease (CMT) type 1A, the most common disease subtype.

The trial was approved in April by South Korea’s Ministry of Food and Drug Safety, which is comparable to the U.S. Food and Drug Administration (FDA). It is now enrolling participants at the Samsung Medical Center.

CMT1A is the most common subtype of CMT, accounting for about two-thirds of all cases. It is caused by a duplication of the PMP22 gene, ultimately resulting in symptoms such as muscle weakness and atrophy of the lower legs beginning in adolescence, followed by hand weakness and decreased sensations later in life.

The disease affects about 8,000 patients in Korea and more than 1.2 million worldwide, but patients still lack curative treatments for the condition.

Engensis is a gene therapy that uses small circular, closed DNA molecules called plasmids to deliver the hepatocyte growth factor (HGF) directly to patients’ cells. HGF is a key protein in the health and development of muscle tissue.

Past research, including other clinical trials, has shown that simple intramuscular injections of this gene therapy have therapeutic potential in nerve regeneration, blood vessel growth, and the prevention of muscular dystrophy. It also appears to suppress pain-related factors in people with neuromuscular disorders.

Engensis is being investigated across a range of indications, including amyotrophic lateral sclerosis, diabetic foot ulcers, diabetic peripheral neuropathy, and heart diseases.

“CMT is similar to diabetic peripheral neuropathy (DPN) in the sense that both diseases are caused by peripheral nerve damage,” Sunyoung Kim, CEO of Helixmith, said in a company press release.

“We hope that Engensis, which has been granted [regenerative medicine advanced therapy] designation by the US FDA and recognized for its potential as a regenerative treatment for DPN, will also be able to provide relief to patients who suffer from this rare disease,” Kim said.

The newly launched Phase 1/2 trial was designed to examine Engensis in 12 CMT1A participants with muscle loss and damage to peripheral nerves (those that transmit messages about movement and sensation back and forth between the limbs and the brain). They will receive Engensis treatments directly in their legs muscles over a 270-day period.

In addition to safety and tolerability, the trial will measure changes in participants’ average daily pain scores at regular intervals, as compared to a placebo treatment.

The Hereditary Neuropathy Foundation (HNF) was among the first conducting research focused on pain management in CMT patients. “We are thrilled to support Helixmith with our research findings and hope that this will be the first disease modifying treatment that addresses pain,” Allison Moore, founder and CEO of the HNF, said in a foundation press release.

Helixmith is headquartered in Seoul, but clinical and developmental facilities are based in San Diego, California. Many of the company’s clinical trials are being conducted in the U.S., and Helixmith also is in preparations to conduct CMT clinical trials in that country.

Forest Ray received his PhD in systems biology from Columbia University, where he developed tools to match drug side effects to other diseases. He has since worked as a journalist and science writer, covering topics from rare diseases to the intersection between environmental science and social justice. He currently lives in Long Beach, California.
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Inês holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in blood vessel biology, blood stem cells, and cancer. Before that, she studied Cell and Molecular Biology at Universidade Nova de Lisboa and worked as a research fellow at Faculdade de Ciências e Tecnologias and Instituto Gulbenkian de Ciência.
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Forest Ray received his PhD in systems biology from Columbia University, where he developed tools to match drug side effects to other diseases. He has since worked as a journalist and science writer, covering topics from rare diseases to the intersection between environmental science and social justice. He currently lives in Long Beach, California.
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