• Disease Progression in DMD

  Diagnosed from symptoms such as unusual walking gait or frequent loss of balance at the age of 3 to 4. Muscle strength weakens over time as the patients age, making their walk difficult. Respiratory or cardiac functions also progressively weakens over time.

  

• The causes of DMD

  The protein called dystrophin is an essential molecule supporting the muscle cell membrane. Without the dystrophin, the muscle cells become damaged and the amount and strength of muscle deteriorate.

  

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

• Major clinical symptoms

  • • Unstable gait.
    • Frequent loss of balance.
    • Large calf muscle.
  • • Difficulty when rising up, needing to put weight on the knee to rise up.
    • Walking on the toes.
    • Difficulty when running or climbing up the stairs.

• Major Biological symptoms

  

• Incidence rate of DMD

  

• Treatments

  Currently available treatment options for DMD are as follow.

  • Physical therapy to help with mobility
  • Corticosteroid to slow down the progression of muscle weakening
  • Treatment for osteoporosis and scoliosis
  • Respiratory support treatment etc

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

• EN001 is a human Wharton's jelly-derived mesenchymal stem cells (WJ-MSCs) based cell therapy

  EN001 is mesenchymal stem cells derived from wharton's jelly (umbilical cord) using proprietary technology developed by
  ENCell Co., Ltd.

  

• 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

Similar to the 8 stem cell therapies that are already in the 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, EN001 was approved for the investigational new drug (IND) by the Ministry of Food and Drug Safety (MFDS) in Korea (2021.05.04).

Therapeutic efficacy of EN001 in DMD

Therapeutic efficacy of EN001 was confirmed in a preclinical trial using mdx mouse, in vivo model for DMD.

• 

  Normal mouse

• 

  DMD mouse

• 

  EN001 Administration

The fibrotic muscle tissues are stained with red (top, sirius red staining), and green (bottom, anti-fibronectin). It was observed that there is more fibrosis occurring in gastrocnemius of the DMD mouse compared compared to the normal mouse.

When EN001 was injected into the DMD mouse, significant reduction of the fibrosis was observed.

• 

  Normal mouse

• 

  DMD mouse

• 

  EN001 Administration

Muscle cells are stained with green (top, anti-Myosin Heavy Chain) and the apoptotic cells are stained with red (bottom, anti-Annexin V). It was observed that there are less normal muscle cells and more apoptotic cells in the DMD mouse compared to the normal mouse.

However, when EN001 was administered to the DMD mouse, an increase in the normal muscle cells (muscle regeneration) and a decrease in the apoptotic cells (anti-apoptosis) were confirmed.