MediciBioㅣ메디치바이오

Avellino corneal dystrophy (ACD) is a serious ophthalmic genetic disorder that causes corneal opacity through a specific mutation in transforming growth factor beta-induced gene (TGFBI) and ultimately leads to blindness. The R124H mutation in TGFBI protein is considered as the disease causing mutation, and appears in 1/870 of Korean population and half of that in the global population. However, no treatment of ACD is approved to date. Our RNAi technology facilitates the selective targeting of the R124H driver mutation of TGFBI at mRNA level, circumventing the off-target effects and enhancing the therapeutic efficacy.

Fuchs endothelial corneal dystrophy (FECD) is a corneal disease with corneal edema, opacity, and visual impairment, caused by deformation and damage of corneal endothelial cells as a result of factors such as UV and oxidative stress. The prevalence of FECD is estimated to be 4~6% in people over the age of 50, and the disease imparts multiple underlying mechanisms and complex pathophysiology. To date, FECD is the main ophthalmic disease requiring corneal transplant surgery, and currently only surgical procedures such as keratectomy are applicable to FECD patients. Our novel FECD treatment strategy suggests blocking both intrinsic and extrinsic pathogenic mechanisms of oxidative stress and fibrosis, and therefore provides effective treatment option for complex FECD pathophysiology.

Myelodysplastic syndrome (MDS) is bone marrow disease characterized by the abnormal production of immature blood cells, and has a high risk of developing into acute myeloid leukemia. Hypomethylation agent (Azacitidine or Decitabine) is currently recommended as the standard therapy for higher MDS; yet, the adapted hypomethylation agent-resistance in over 50% patients becomes a recurrent clinical problem in treating MDS. MediciBIO develops a novel oral hypomethylation agent that overcomes the current drug-resistance, and aims to improve patient convenience of medication as well as therapeutic outcomes of MDS patients.

Avellino corneal dystrophy (ACD) is a serious ophthalmic genetic disorder that causes corneal opacity through a specific mutation in transforming growth factor beta-induced gene (TGFBI) and ultimately leads to blindness. The R124H mutation in TGFBI protein is considered as the disease causing mutation, and appears in 1/870 of Korean population and half of that in the global population. However, no treatment of ACD is approved to date. Our RNAi technology facilitates the selective targeting of the R124H driver mutation of TGFBI at mRNA level, circumventing the off-target effects and enhancing the therapeutic efficacy.

Fuchs endothelial corneal dystrophy (FECD) is a corneal disease with corneal edema, opacity, and visual impairment, caused by deformation and damage of corneal endothelial cells as a result of factors such as UV and oxidative stress. The prevalence of FECD is estimated to be 4~6% in people over the age of 50, and the disease imparts multiple underlying mechanisms and complex pathophysiology. To date, FECD is the main ophthalmic disease requiring corneal transplant surgery, and currently only surgical procedures such as keratectomy are applicable to FECD patients. Our novel FECD treatment strategy suggests blocking both intrinsic and extrinsic pathogenic mechanisms of oxidative stress and fibrosis, and therefore provides effective treatment option for complex FECD pathophysiology.

Myelodysplastic syndrome (MDS) is bone marrow disease characterized by the abnormal production of immature blood cells, and has a high risk of developing into acute myeloid leukemia. Hypomethylation agent (Azacitidine or Decitabine) is currently recommended as the standard therapy for higher MDS; yet, the adapted hypomethylation agent-resistance in over 50% patients becomes a recurrent clinical problem in treating MDS. MediciBIO develops a novel oral hypomethylation agent that overcomes the current drug-resistance, and aims to improve patient convenience of medication as well as therapeutic outcomes of MDS patients.

PIPELINE

MDC-101

Gene Therapy for Avellino Corneal Dystrophy

MDC-201

Therapy for Fuchs Endothelial Corneal Dystrophy

MDC-202

Therapy for Myelodysplastic Syndrome

MDC-101: Gene Therapy for Avellino Corneal Dystrophy

MDC-201 : Therapy for Fuchs Endothelial Corneal Dystrophy

MDC-202 : Therapy for Myelodysplastic Syndrome