ENCell Registers EN001 Use Patent for Charcot-Marie-Tooth Disease Treatment in Australia

ENCell, a company specializing in advanced biopharmaceutical CDMO (Contract Development and Manufacturing Organization) and new drug development, announced that its patent for the prevention and treatment of Charcot-Marie-Tooth disease (CMT) using insulin secreted from mesenchymal stem cells EN001 or MSC has been officially registered with the Australian Patent Office.

CMT is a genetic disorder that causes deformities of the hands and feet and muscle atrophy, and in severe cases, can even lead to loss of vision and hearing. Despite being a rare disease with a relatively high incidence rate, there are currently no approved treatments, making it a significant challenge for both patients and medical professionals.

The recently registered EN001 use patent pertains to a new CMT treatment strategy that combines mesenchymal stem cells, which have demonstrated neuroprotective and regenerative properties in various studies, with insulin, which plays a critical role in metabolic regulation and maintenance of neural function. This patent was previously registered in Europe last year.

An ENCell representative stated, "We plan to continue expanding patent registrations in major global markets, including Europe and Australia, to secure ENCell's exclusive rights," adding, "This use patent is expected to be an important foundation for collaboration with global pharmaceutical companies and technology exports in the field of rare disease therapeutics."

ENCell's new drug pipeline EN001, under development for CMT treatment, is a mesenchymal stem cell therapy cultivated using ENCell's proprietary ENCT (ENCell Technology) platform. It is characterized by its ability to suppress cellular aging and secrete higher amounts of therapeutic substances. This therapy migrates to damaged nerves, releases therapeutic substances, and helps regenerate nerve myelin.

In February of this year, it was designated as an Orphan Drug by the U.S. FDA. Notably, since CMT is classified as a rare disease, conditional marketing authorization can be applied for after the completion of Phase 2 clinical trials. As EN001 is about to enter Phase 2a clinical trials, there has been a growing number of inquiries from CMT patient associations worldwide regarding the clinical status and participation in EN001 trials, reflecting heightened expectations for new rare disease treatments.