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• Charcot-Marie-Tooth disease, CMT

  Charcot-Marie-Tooth disease is a hereditary neuropathy characterized by abnormalities in the motor and sensory functions caused by damage in the myelin sheath that surrounds nerves.

• Disease Progression in CMT

  The onset period of the symptoms differs by individual, and the patients experience gradual atrophy, weakness, and loss of sensations in the foot and hand muscles.
  The severity of the symptoms ranges from very mild condition with no difficulties in daily life to needing a wheelchair.

  

• The causes of CMT

  There have been approximately 70 causative genes implicated in CMT so far. However, in about 50% of the patients, the causative genes have not been identified yet. In order for the nerves to function normally, they need an insulating lipid layer called myelin sheath.
  CMT is caused by the damages in myelin sheath.

  

The major symptoms suffered by the CMT patients are as follow.

• Major clinical symptoms

  • Muscle weakness starting from the lower extremities.
  • Lack of sensation such as pain and warmth in hands and feet.
  • Wasting of the calf muscle. (stork leg)
  • Cavus foot (foot with high arch)and hammer toe (toe curled in a Z-shape)

• Major biological symptoms

  

• Prevalence of DMD

  

• Treatments

  The currently available treatment options for CMT are as follow.

  • Assistive Devices to prevent foot drop
  • Physiotherapy or occupational therapy
  • Drugs that slow the disease progression (e.g. ascorbic acid, PTX3003 etc.)

  Recently, there have also been several attempts with clinical trials on gene therapy using gene editing.

Safety of EN001

Similar to the 8 stem cell therapies that are already in the global market, EN001 is also mesenchymal stem cell.
The safety of mesenchymal stem cell has been confirmed in over 8,000 cases of clinical trials conducted worldwide, and not a single serious adverse event has been reported so far.

EN001, developed solely by ENCell Co., Ltd., has been confirmed for its safety through a thorough verification by the GLP certified testing organization (KIT, Korea Institute of Toxicology). Based on this GLP toxicity report, ENCell Co., Ltd. has obtained an investigational new drug (IND) approval by the Ministry of Food and Drug Safety (MFDS) in Korea (2021.06.29).

Therapeutic efficacy of EN001 in CMT

Therapeutic efficacy of EN001 was confirmed in a preclinical trial using C3 mouse, a mouse model for CMT.

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  Normal mouse

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  CMT mouse

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  EN001 Administration

The myelin sheath surrounding the nerves are stained in dark grey. It was observed that the myelin sheath is thinner and unstable in CMT mouse compared to the normal mouse.

However, when EN001 was administered to the CMT mouse, thicker and healthier myelin sheath were observed.

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  Normal mouse

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  CMT mouse

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  EN001 Administration

Healthy muscle cells are stained in green. It was observed that there are less healthy muscle cells in the CMT mouse compared to the normal mouse.

However, when EN001 was administered to the CMT mouse, an increase in the healthy cells (regeneration of muscle cells) was confirmed.