GENIXCURE
Addressing the Specific Cell and Gene Therapy Needs

GENIXCURE is an engineering biotech firm from South Korea that develops novel Adeno-Associated Virus (AAV) vectors powered by its own AI system called InsightMiner.

By integrating AI technology with traditional methods of directed evolution for AAV, GENIXCURE accelerates the identification and optimization of AAV vector candidates. This approach enhances the transduction efficiency and improves de-targeting capabilities of the vectors, streamlining the discovery and development process for advanced gene therapy solutions.

At its core, the company’s cutting-edge CDMO services and proprietary Cell-specific AAV Research Engine (CARE) platform address specific cell and gene therapy needs. The company also has its wholly-owned therapeutic programs against ALS, FTD and Lafora disease.

“Powered by InsightMiner, the CARE platform explores novel AAV vectors with enhanced target specificity and transduction efficiency, which suggests amino acid sequences that need to be inserted into certain regions of the AAV cap gene,” says Moon Hong-Sung, CEO of GENIXCURE.

Since its inception in early 2021, GENIXCURE's AI system, InsightMiner, has become adept at suggesting typical sequences for the CARE platform to validate lead vectors more efficiently than any other AI systems from competing biopharmaceutical companies. InsightMiner focuses on AAV capsid engineering, utilizing a machine learning approach to optimize its process.

The CARE platform aims to reduce targeting of certain tissues or cells, particularly the liver, which is a common concern for pharmaceutical companies conducting AAV gene therapy programs.

These programs often face safety challenges during clinical trials, including liver toxicity in higher dose groups. GENIXCURE's liver-detargeting vector candidates transduce fewer liver cells, providing invaluable support to clinical trial sponsors by improving safety outcomes.

XOB-031 is the most advanced lead vector from GENIXCURE's CARE platform, showcasing superior transduction capabilities in brain cells compared to wildtype AAV9. The company has a growing portfolio of candidates for lead vectors that demonstrate enhanced targeting performance across various cell types, including muscle, hepatocyte, and Schwann cells. These developments signify GENIXCURE's ongoing commitment to advancing gene therapy through specialized vector engineering.

Leveraging InsightMiner, GENIXCURE skips a laborious and time-consuming procedure of library expansion. In the future, every biotech firm engaging in AAV capsid engineering would have no choice but to repeat the experimental cycle using an animal model, normally mice, to find improved variants no matter how miscellaneous it is. GENIXCURE uses InsightMiner at this stage to get practical candidates. In addition to the 'on- & de-targeting feature,' GENIXCURE is upgrading InsightMiner to challenge commercial vectors evading the immune system more effectively, penetrating BBB better, and making manufacturing easier.

Among its therapeutic programs from GXC-301 to GXC- 304, GXC-301 is the most progressive asset in terms of developmental status. Currently, GENIXCURE is applying to ODD and RPDD and expects to be successful by September 2024, the sunset timeline of the RPDD voucher from the FDA.

“We are placed as a unique biotech firm due to our AI-screening platform for novel AAV vectors, and we welcome partners for R&D collaboration, BD and investment,” says Moon.