Pipeline
Cell Therapy Medication Pipeline
Division Division clinical
  • Research
  • Preclinical
  • Phase 1/2a
  • Phase 2b
  • Phase 3
Voiding Dysfunction Interstitial Cystitis

Orphan Drug Designation

Approval
Underactive Bladder(UB)
Overactive Bladder (OB)
Autoimmune Disorder Atopic Dermatitis
Urticaria
Musculoskeletal Sports Injuries
Neurological Dementia
Non-Medication Pipeline
Division
  • Research
  • Preclinical
  • Commercialization
 Notes
AD Pig
Development Complete, Mass Production
Bio-derived Active Substances
Development and License Complete, Mass Production
Voiding Dysfunction
Interstitial Cystitis Treatment (MR-MC-01)

Severe inflammatory disorder of unknown etiology that causes bladder tissue damage with over 90% of the patients being female. According to Ministry of Food and Drug Safety Notice No. 30 published on November 12, 2020, this was designated as an orphan drug in the development stage.

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Interstitial Cystitis and MR-MC-01
  • Symptoms include abnormal sensory urgency, frequent urination, and bladder pain. The exact etiology is unknown, and over 90% of those affected are female.
  • There are no medical or surgical treatments that provide complete relief, and medications that are used as adjuvant and currently on the market include those that have dimethyl sulfoxide (DMSO) and pentosan polysulfate sodium (PPS) as their main ingredients.
  • It is classified as a rare condition in most countries, and it affects approximately 5-7 million people worldwide
    (5 million in the United States). The estimated market size is USD 402 million (2022).
  • Efficacy of cell therapy was proved using acute (type 2) and chronic (type 1) disease animal models
  • Frequent urination, which is the main symptom of interstitial cystitis, improved to normal levels. Urination amount, bladder volume, residual urine volume, bladder contractility during urination, etc. were also shown to have improved on the bladder function index.
  • Credibility obtained through publication in international journals
Approval of Clinical Trial Plan and Designation of Development Stage Orphan Drug
  • IIT approved by the Ministry of Food and Drug Safety on interstitial cystitis (No. 32855)
  • SIT approved by the Ministry of Food and Drug Safety on interstitial cystitis (No. 33308)
  • MR-MC-01, designation of development stage orphan drug (No. 30)
  • Approval of IIT plan (No. 32855)
  • Approval of SIT plan (No. 33308)
  • Designation of development stage orphan drug (No. 30)
Safety and Efficacy of MR-MC-01
  • Through testing at specialized institutions and certifying institutions, it has been proven that MR-MC-01 has no or extremely low immunogenicity.
  • Through the GLP certification institution, it was certified as safe on single-dose toxicity, immune toxicity, tumorigenicity and the evaluation of biodistribution tests
  • It has been proven to be effective in various urination disorders in animal models.
  • Treatment potential was shown for inflammatory and autoimmune skin disorders in animal models.
Safety of MR-MC-01 Approved by External Organizations
  • Immunogenicity
  • Single-Dose Toxicity
  • Tumorigenicity
  • Biodistribution
A Variety of Animal Models Used to Evaluate the Efficacy of MR-MC-01 in Interstitial Cystitis
  • Acute Model

    Hydrogen chloride (HCl)-induced interstitial cystitis model I Sci Rep. 2017 Aug 21;7(1):8872

  • Acute Model

    Ketamine-induced interstitial cystitis model I Int Neurourol J. 2018;22

  • Chronic Model

    Lipopolysaccharide (LPS)-induced interstitial cystitis model I Theranostics. 2018 Nov 9;8(20):5610-5624

Efficacy Evaluation of MR-MC-01 in Interstitial Cystitis Animal Models (Non-Clinical)
  • Normal Animal Group (Sham)
  • Chronic Interstitial Cystitis Animal Group (LPS)
  • Cell Therapy Animal Group (MR-MC-01)
    • 0.35
    • 0.15
    • 0.26
    Urination Amount
    • 0.66
    • 0.18
    • 0.36
    Bladder Volume
    • 0.31
    • 0.03
    • .0.1
    Residual Volume
    • 98.29
    • 27.42
    • 54.29
    Urination Interval
    • 53.29
    • 95.86
    • 66.85
    Urination Pressure
    • 31
    • 60.95
    • 37.25
    Base Pressure
    • 53.43
    • 97.17
    • 67.07
    Maximum Pressure
Efficacy Comparison of MR-MC-01 to Market Medications
  • A non-clinical trial was performed to compare the efficacy of MR-MC-01 to US FDA-approved DMSO-based medications.
  • Functional improvement of the bladder and pathological improvement were both confirmed for MR-MC-01, and it was shown to be superior to DMSO.
  • Normal Animal Group (Sham)
  • Chronic Interstitial Cystitis Animal Group (LPS)
  • Cell Therapy Animal Group (MR-MC-01)
  • Medication with Approved Ingredient Administration Animal Group (DMSO)
    • 102.8
    • 15.94
    • 72.2
    • 45.69
    Urination Interval
    • 53.47
    • 93.82
    • 59.3
    • 79.49
    Urination Pressure
    • 28.15
    • 55.76
    • 31.96
    • 36.26
    Base Pressure
    • 53.53
    • 92.63
    • 59.36
    • 79.71
    Maximum Pressure
    • 0.37
    • 0.07
    • 0.34
    • 0.22
    Urination Amount
    • 0.35
    • 0.15
    • 0.26
    • 0.22
    Bladder Volume
    • 0.32
    • 0.04
    • 0.14
    • 0.09
    Residual Volume
Mechanism of Action
  • After the administration of MR-MC-01 it was found through bladder tissue analysis that the drug inhibits apoptosis, decreases fibrosis caused by inflammation and reduces the infiltration of immune cells, which are inflammatory cells.
  • Wnt and IGF signal activation regulation was confirmed through molecular level analysis as the pharmacological effect
  • Normalization was confirmed in bladder inflammation-related gene expression analysis after the administration of MR-MC-01
  • Anti-apoptosis
    • Normal
    • Interstitial Cystitis
    • Cell Treatment MR-MC-01
    • 0.64
    • 6.96
    • 1.25
  • Anti-fibrosis
    • Normal
    • Interstitial Cystitis
    • Cell Treatment MR-MC-01
    • 4.31
    • 6.03
    • 4.54
  • Inhibition of the infiltration of mast cells
    • Normal
    • Interstitial Cystitis
    • Cell Treatment MR-MC-01
    • 0.98
    • 4.86
    • 1.3
  • Wnt - IGF cell signal activation
      • 65
      • 42
      • 45
      Urination Pressure
      • 62
      • 30
      • 33
      Urination Interval
      • 0.25
      • 0.14
      • 0.12
      Urination Amount
      • 0.42
      • 0.19
      • 0.22
      Bladder Volume
      • 0
      • 7.5
      • 4.2
      Non-Urination Contraction

    • MMSC+PBS

    • MMSC+ Gefitinib

    • MMSC+ Indomethacin

  • Expression of genes related to bladder function improvement
Underactive Bladder (UB)

This is a disorder where urination does not happen even when the bladder is full due to the decreased function of the bladder muscles from aging or nerve damage. It is estimated that the number of patients will increase due to the aging population.

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UB and MMSC
  • This is characterized by a mixed group of symptoms that suggest decreased detrusor contractions such as urinary retention and urination time delay, accompanied by delayed urination and weak urine.
  • It is reported to be related to aging, and its prevalence is reported to increase with age, affecting 48% of patients over the age of 70.
  • There are no fundamental treatments, and currently, drug therapy with catheterization is performed to force urination. However, risks of complications exist such as urinary tract injury, infections, etc.
  • After administration of MR-MC-01 to ischemic and diabetic underactive bladder disorder models, improvement of bladder function such as voiding volume, bladder volume, residual urine volume, and bladder pressure were verified.
  • Credibility obtained through publication in international journals
UB and MMSC
  • Animal Model 1.

    Ischemic underactive bladder model I J Clin Med. 2020 Sep 3;9(9):2853

  • Animal Model 2.

    Diabetic underactive bladder model

Efficacy Evaluation of MMSC in Underactive Bladder Animal Model (Non-Clinical)
  • Normal Animal Group
  • Diabetic Underactive Bladder Animal Group
  • Cell Therapy Animal Group (MMSC)
    • 0.35
    • 0.15
    • 0.26
    Urination Amount
    • 0.66
    • 0.18
    • 0.36
    Bladder Volume
    • 0.31
    • 0.03
    • .0.1
    Residual Volume
    • 98.29
    • 27.42
    • 54.29
    Urination Interval
    • 53.29
    • 95.86
    • 66.85
    Urination Pressure
    • 31
    • 60.95
    • 37.25
    Base Pressure
    • 53.43
    • 97.17
    • 67.07
    Maximum Pressure
Mechanism of Action
  • Analysis of reactivity to KCl and EFS (electrical stimulation test) showed improved reactivity in the MR-MC-01 administration group
  • Confirmed muscle contraction through ATP and carbachol concentration-dependent reactivity
  • Confirmed engraftment of transplanted cells and conversion to myocytes via tissue analysis
  • Normalization was confirmed in bladder muscle contraction-related gene expression analysis after the administration of MR-MC-01
  • Contraction to KCI and reactivity to EFS
  • Contraction to 1mM ATP and reactivity to carbachol concentration
  • Engraftment of transplanted MMSC and conversion to muscle cells
    • a-SMA/GFP
      DM+M-MSC
      (muscle)
    • Ki-67
    • Ki-67
    • DM+M-MSC
      (pericyte)
    • DAPI (Nuelear)
      DM+M-MSC
      (a-SMA/GFP)
    • GFP (M-MSC)
    • Tissue markers
    • Merged
    • DM+M-MSC
      (Desmin/GFP)
  • Confirmation of expression of genes related to muscle contraction
Overactive Bladder (OB)

This is a disorder characterized by abnormal contraction of the detrusor muscle due to various reasons causing incontinence regardless of the patient’s urge to urinate.
It has been evaluated that the number of sufferers of this disease is increasing especially in the younger generation.

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OB and MMSC
  • Urinary urgency (symptom where one cannot hold one’s urine when the urge to urinate is felt) is the main symptom. It is usually accompanied by frequent urination, nocturia, and also can be accompanied by urge incontinence.
  • The prevalence increases with age, and it is known to be affecting three out of every ten elderly people over the age of 65, and over 700 million people worldwide.
  • Company A has produced drug B, but it has been shown to be conservative in efficacy and prone to causing side effects.
  • The estimated market size is USD 3.62 billion in 2017 and is expected to grow to USD 5.86 billion by 2025.
  • Procurement of a method for establishing an animal model that can represent actual clinical status
Establishment of an overactive bladder animal model that can represent actual clinical status
  • Sham
  • MR-MC-01

Stem Cells Dev. 2014 Mar 15;23(6):654-63

    • 200
    • 40
    Urination interval
    • 32
    • 29
    Urination Pressure
    • 0.1
    • 0.4
    Bladder volume
    • 1
    • 0.6
    Residual volume
Neurological Disorders
AD Treatment

This is the most common degenerative brain disease that causes dementia. Cognitive dysfunction and behavioral disorders are caused by the apoptosis of brain cells, which is caused by the deposition of beta amyloids and phosphorylation of Tau proteins.

Autoimmune Disorders
Atopic Dermatitis/Urticaria Treatment

This is a skin disorder with various etiology and has been thought to be linked to the immune system. MMSC can decrease mast cells effectively, suppress immune reactions through immune regulation, and can be used in patients who do not respond to omalizumab and antihistamines.

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Inflammatory/Autoimmune Skin Disorders and MMSC
  • Atopic dermatitis, a type of inflammatory/autoimmune skin disorder, is the most common pediatric skin disorders, which is incurable for humans in the 21st century.
  • Intractable skin disorders with chronic recurrence and severe pruritus
  • There are hundreds of millions of patients worldwide, and large pharmaceutical companies and biotech companies are competing to develop therapeutics
  • The market size is estimated to be USD 5.63 billion in 2022.
Efficacy evaluation of MMSC-cell secretion in atopic dermatitis animal model (non-clinical)
  • Registration for application patent completed
  • Currently obtaining data on safety and efficacy in the non-clinical stage
  • Establishing a development strategy through consultation with renowned domestic clinical experts
Biomedicines. 2020 Oct 21;8(10):439
  • Atopic dermatitis animal group (DNCB) normal
  • cell secretion treatment group
  • 0.2
    IL-1b
  • 0.7
    IL-4
  • 0.4
    IL-6
  • 0.4
    IL-10
  • 0.5
    IL-13
  • 0.7
    Ig E

[AP model (DNCB) measure value standards]

  • Normal animal group
  • Atopic dermatitis animal group
  • Cell secretion treatment group

[Skin thickness, H&E, 10 x]

[Mast cell number, Toluidine blue, 20 x]

Musculoskeletal Disorders
Sports injuries (rotator cuff tear)

MMSC’s ability to suppress inflammation and promote tissue regeneration can greatly contribute to recovery from body tissue injuries. The efficacy of MMSC has been proven in rotator-cuff-tear animal models.

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Production and evaluation of disease animal models

* Ligament resection of supraspinatus and infraspinatus muscle

  • 8 weeks of monitoring in total
  • Histological analysis
  • Inflammation related cytokine analysis
  • Function evaluation
Histological analysis – Inhibition of tissue fiber fibrosis, reduction of inflammation cells and tissue regeneration effect confirmed
  • Supraspinatus muscle
    • Control
    • MMSC
    • 24 h
    • 72 h
    • 7-d
    • 4 wks
    • 8wks
  • Infraspinatus muscle
    • Control
    • MMSC
  • Supraspinatus muscle
    • Control
    • MMSC
    • 24 h
    • 72 h
    • 7-d
    • 4 wks
    • 8wks
  • Infraspinatus muscle
    • Control
    • MMSC
  • Supraspinatus muscle
    • Control
    • MMSC
    • 24 h
    • 72 h
    • 7-d
    • 4 wks
    • 8wks
  • Infraspinatus muscle
    • Control
    • MMSC
Inflammation related cytokine analysis – Reduction of intracellular inflammatory cytokines in tissues confirmed