Therapeutics R&D Advanced cell / gene-based biopharmaceutical development and CDMO
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
Major biological symptoms
Prevalence of DMD
Treatments
EN001 is a human Wharton's jelly-derived
mesenchymal stem cells (WJ-MSCs)
based cell therapy developed by
ENCell Co., Ltd. for CMT patients.
EN001 is a human Wharton's jelly-derived mesenchymal stem cells (WJ-MSCs) based cell therapy.
Mesenchymal Stem Cells
Mesenchymal stem cell is a type of adult stem cell derived from tissues such as umbilical cord, bone marrow and adipose tissue.
Mesenchymal stem cells are already used as treatments for myocardial infarction, osteoarthritis, amyotrophic lateral sclerosis etc.
There are in total 8 types of mesenchymal stem cell based therapies in the market and they have shown high safety profile.
Safety of EN001
Therapeutic efficacy of EN001 in CMT
Therapeutic efficacy of EN001 was confirmed in a preclinical trial using C3 mouse, a mouse model for CMT.
Normal mouse
CMT mouse
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.
Normal mouse
CMT mouse
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.