Hong-Sung Moon, CEO of GENIXCURE
Delivering desired genes to target sites using adeno-associated viruses (AAVs), which are harmless to humans
AAV gene delivery technology is expected to achieve equivalent therapeutic effects at lower doses than competing groups
Its flagship product is the ‘Next-Generation AAV Vector Platform.’ AAV vectors are harmless to humans and enable precise gene delivery, establishing themselves as core technology for global gene therapies. GENIXCURE is further evolving this technology by developing a next-generation platform that achieves high delivery efficiency even at low doses.
The company is developing next-generation AAV vectors using InsightMiner™, its proprietary, machine-learning-based AAV engineering platform. Unlike conventional methods, GENIXCURE’s unique InsightMiner™ platform combines vast human-derived big data with AI to precisely design human cell-specific capsids with high clinical applicability.
The flagship vector XOB-031_v2, developed through this platform, exhibits approximately 450-fold enhanced central nervous system (CNS) targeting compared to conventional wild-type AAV9. It overcomes the limitations of existing AAV9-based therapeutics and is recognized as a strategic pipeline capable of providing alternatives to enable new drug development or improve existing therapies for Korean and international biopharmaceutical companies.
Currently, many global companies face challenges in AAV clinical trials due to delivery efficiency and toxicity issues. GENIXCURE is expanding domestic and international partnerships through plasmid/vector supply and joint development based on its high-efficiency vector technology that addresses these challenges.
“AAV is a core platform changing the paradigm of gene therapy. GENIXCURE will elevate delivery efficiency and safety to the next level with AI-based technology, driving meaningful change in the global gene therapy development market.”
Moon said “Over half of the gene therapies approved by the FDA for in vivo use utilize AAV vectors and added that AAV is projected to dominate the majority of the viral gene vector market going forward.”
Recently, a growing number of Korean pharmaceutical companies and biotech firms have adopted GENIXCURE vectors for their gene therapy development. In particular, the major Korean pharmaceutical company C and the gene therapy specialist N have signed material transfer agreements (MTAs) with GENIXCURE and are currently conducting vector performance tests. Additionally, GENIXCURE is pursuing collaborative joint development research with the KOSDAQ-listed company E and the gene therapy developer G, and it has signed a joint development research agreement with DongKoo Bio&Pharma for a gene therapy utilizing its vector. Having previously licensed out a portion of the equity in GENIXCURE’s latest vector, XOB-031_v2, the value of the technology and its business scalability have been demonstrated.
Regarding future plans, Moon stated, “GENIXCURE views the acquisition of non-human primate experimental data for its AAV vector, scheduled for the second half of this year, as a crucial turning point,” adding, “Based on this, we will further expand discussions on joint development and technology transfer with global pharmaceutical companies.” He added, “Furthermore, we aim to substantiate the non-clinical achievements of our next-generation vector platform with data, prepare for future clinical entry, and simultaneously strengthen global partnerships to drive tangible commercialization outcomes.”
Founded: April 2019
Main business: AI-based AAV vector platform development
Achievements: Selected for the 2024 Global Unicorn Project ‘2024 Baby Unicorn Project’; GXC-301 designated as rare pediatric disease designation (RPDD) and orphan drug designation (ODD) by the U.S. FDA; Selected for the 2024 SME Technology Innovation Development Project ‘Market Expansion Type’ (Ministry of SMEs and Startups); Registered patent for Lafora disease treatment candidate GXC301; Selected for the 2025 Startup Scale-up Package project (Ministry of SMEs and Startups); Selected for the 2025 SME Technology Innovation Development Project ‘Market Expansion Type’ project (Ministry of SMEs and Startups); Signed a joint R&D agreement with DongKoo Bio&Pharma for AAV gene therapy; Filed an application for an AAV vector patent covering CNS-specific peptides and AAV variants
reporter jinho lee
jinho2323@hankyung.com
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