PIPELINE

Renaissance of Medicine

PIPELINE

Renaissance of Medicine

MDC-101
Gene Therapy for Avellino Corneal Dystrophy

Anti-sense oligonucleotide for targeting mutant TGFBI R124H mRNA which causes the precipitation of mutant TGFBI and corneal turbidity

Progress in Retinal and Eye Research 50 (2016) 67-88


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 selectively targets mutant TGFBI mRNA, minimizing off-target effects. In a TGFBI mutant animal model, it also demonstrates the potential of a safe and convenient therapeutic option by effectively suppressing the expression of the mutant gene without causing ocular irritation, through topical administration in the form of eye drops. 

MDC-301
H5Nx Avian Influenza Vaccine

Capless Self-Amplifying mRNA (CLsaRNA) Vaccine for H5Nx Avian Influenza using Proprietary LNP Technology

Highly pathogenic avian influenza (HPAI), caused by Clade 2.3.4.4.b H5N1 and its variants (H5Nx), is a severe viral infection that poses a significant threat to both global poultry industries and human health. MDC-301 is an mRNA vaccine targeting these circulating strains of HPAI. Compared to conventional mRNA vaccines, it utilizes capless self-amplifying mRNA combined with a localized delivery LNP platform to improve both efficacy and safety. The vaccine has demonstrated strong neutralizing antibody responses in animal models and reduces systemic reactogenicity through localized delivery. The technology is fully protected by proprietary patents and enables cost-effective manufacturing by eliminating the need for expensive materials such as Cap and modified uridine.

MDC-101 : Gene Therapy for Avellino Corneal Dystrophy

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 selectively targets mutant TGFBI mRNA, minimizing off-target effects. In a TGFBI mutant animal model, it also demonstrates the potential of a safe and convenient therapeutic option by effectively suppressing the expression of the mutant gene without causing ocular irritation, through topical administration in the form of eye drops. 

MDC-301 : H5Nx Avian Influenza Vaccine

Highly pathogenic avian influenza (HPAI), caused by Clade 2.3.4.4.b H5N1 and its variants (H5Nx), is a severe viral infection that poses a significant threat to both global poultry industries and human health. MDC-301 is an mRNA vaccine targeting these circulating strains of HPAI. Compared to conventional mRNA vaccines, it utilizes capless self-amplifying mRNA combined with a localized delivery LNP platform to improve both efficacy and safety. The vaccine has demonstrated strong neutralizing antibody responses in animal models and reduces systemic reactogenicity through localized delivery. The technology is fully protected by proprietary patents and enables cost-effective manufacturing by eliminating the need for expensive materials such as Cap and modified uridine.

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