Efficacy-enhancing and long-acting technology for siRNA or ASO drugs by harnessing albumin with lipid-conjugated oligopeptide, "Medici Peptide"

Oligonucleotide-based or peptide-based drugs are rapidly eliminated from blood circulation after administration, attributable to their short in vivo half-lives; therefore, large amount of drug administration is necessary to show sufficient in vivo efficacy.  Our Medici Peptide is developed to efficiently bind to albumin in vivo, and Medici Peptide-Drug Conjugate (MDC) can therefore increase retention time of oligonucleotide or peptide in blood circulation resulting in enhanced efficacy, and further maintain long-term in vivo efficacy by steady release of drugs from the biodegradable linker.

Novel biodegradable ionizable lipid, “Medici Ionizable Lipid” which enables lipid nanoparticles containing gene therapeutics to show excellent efficacy and safety

Development of COVID-19 vaccine served as a momentum to pay attention to an ionizable lipid which is a core component of lipid nanoparticle of mRNA COVID-19 vaccine, as a clinically proven carrier. Although mRNA vaccine market has grown explosively, there is still insufficient supply of carrier technology to fulfill the increasing needs for gene therapeutics. Development of mRNA medicine is also limited due to patent monopoly of biodegradable ionizable lipid carriers including ALC-0315 and SM-102 until 2036. Medici Ionizable Lipid is a novel biodegradable ionizable lipid that avoids the existing patents of ionizable lipids or lipid nanoparticles, which can contribute to development of new gene therapeutics with excellent efficacy and safety. 

The LNP composed of Medici Ionizable lipids is differentiated into two types for systemic and local gene delivery, respectively.

• Systemic delivery : It exhibits superior gene delivery efficacy after Intravenous (IV) administration in comparison to LNPs composed of a FDA approved ionizable lipids. Notably, it also shows significantly lower and minimal immunogenicity and inflammation profiles, addressing a limitation in currently existing ionizable lipids that restricts repeated administration due to carrier immunogenicity. This enables the development of RNA therapeutics that require both multiple and frequent repeated administrations. 
• Local delivery : It demonstrates favorable gene delivery efficacy in muscle with a significant reduction in undesired systemic exposure after Intramuscular (IM) administration, whereas currently existing ionizable lipids have a disadvantage of systemic exposure after IM injection, causing systemic reactogenicity. Therefore, LNPs composed of Medici ionizable lipids allow for the development of infectious disease vaccines as well as cancer vaccines due to their locally restricted characteristics after injection. 

Novel ionizable lipid, "Medici Targeting Lipid" which enables lipid nanoparticles containing gene therapeutics to distribute into specific tissues by targeting transporters or proteins highly expressed in the tissues

Not limited to vaccines for infectious diseases, gene therapy has been expanding its scope of development to cancer vaccine, immunotherapy and intractable neuronal disease treatment. However, no distinct non-viral carriers have been developed for targeting other tissues except liver, such as cancer, lung or brain tissues. Therefore, targeted gene therapy shows a great promise as next-generation therapeutics in the field of gene therapy. Our Medici Targeting Lipid enables the delivery of gene-containing lipid nanoparticle to specific tissues including liver, lung, and cancer, contributing to the development of gene therapeutics for diverse clinical indications.

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