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.

A vaccine targeting Clade 2.3.4.4b H5N1 and H5Nx, the current causes of highly pathogenic avian influenza, is being developed by integrating capless self-amplifying mRNA technology with local delivery LNP technology.

In terms of efficacy, the vaccine has demonstrated superior performance compared to conventional mRNA vaccines in mouse and ferret models, as evidenced by its ability to induce neutralizing antibodies and protect against viral challenge. Regarding safety, the local delivery mechanism minimizes systemic distribution, ensuring that the vaccine is delivered only to the intended site. This approach helps reduce excessive systemic inflammatory responses (reactogenicity) and associated adverse effects.

Moreover, this technology is fully protected by proprietary patents covering both the RNA and LNP core components, allowing for unrestricted commercial utilization. Additionally, by eliminating the need for expensive raw materials such as Cap and modified uridine, production costs can be significantly reduced, making this vaccine highly cost-effective and widely applicable with strong market competitiveness.

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 oligonucleotide drug 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.

A vaccine targeting Clade 2.3.4.4b H5N1 and H5Nx, the current causes of highly pathogenic avian influenza, is being developed by integrating capless self-amplifying mRNA technology with local delivery LNP technology.

In terms of efficacy, the vaccine has demonstrated superior performance compared to conventional mRNA vaccines in mouse and ferret models, as evidenced by its ability to induce neutralizing antibodies and protect against viral challenge. Regarding safety, the local delivery mechanism minimizes systemic distribution, ensuring that the vaccine is delivered only to the intended site. This approach helps reduce excessive systemic inflammatory responses (reactogenicity) and associated adverse effects.

Moreover, this technology is fully protected by proprietary patents covering both the RNA and LNP core components, allowing for unrestricted commercial utilization. Additionally, by eliminating the need for expensive raw materials such as Cap and modified uridine, production costs can be significantly reduced, making this vaccine highly cost-effective and widely applicable with strong market competitiveness.

PIPELINE

MDC-101

Gene Therapy for Avellino Corneal Dystrophy

MDC-201

Therapy for Fuchs Endothelial Corneal Dystrophy

MDC-301

H5Nx Avian Influenza Vaccine

MDC-101: Gene Therapy for Avellino Corneal Dystrophy

MDC-201 : Therapy for Fuchs Endothelial Corneal Dystrophy

MDC-301 : H5Nx Avian Influenza Vaccine