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Signed a MOU with Modalis Therapeutics Corporation, jointly developing a XOB031-mediated AAV gene therapy for Alzheimer’s disease
2024-07-12
Genixcure Signs MOU with Modalis for Joint Development of AAV Gene Therapy Using Proprietary AAV Vector
Genixcure Inc.(CEO Hong-Sung Moon), a company specializing in AI-based AAV engineering platforms (CARETM), announced that it has signed a strategic memorandum of understanding (MOU) with Modalis Therapeutics Corporation (Modalis, www.modalistx.com), a Tokyo Stock Exchange-listed company, to jointly develop a new AAV gene therapy targeting Alzheimer's disease using its proprietary CNS-specific vector, 'XOB-031'.
Modalis (Tokyo Stock Exchange: 4883), founded in 2016 based on technology from the University of Tokyo, is a global leader in AAV gene therapy development, with a corporate research lab in Waltham, Massachusetts. Modalis develops unique gene therapies that modulate the switches of pathology-related targets using its proprietary epigenome editing technology, CRISPR-GNDM® (Guide Nucleotide-Directed Modulation). The company’s pipeline focuses primarily on muscle and CNS diseases, utilizing AAV as the delivery vehicle for all its therapies.
GENIXCURE, founded in 2019, has its headquarters and R&D center in Suwon, South Korea, and develops gene therapies using its proprietary AAV vectors from the AI-based AAV Capsid Engineering Platform, CARETM (Cell-specific AAV Research Engine), It also develops AAV gene therapies for the treatment of CNS disease such as Alzheimer’s disease, ALS, Lafora Disease, and Frontotemporal Dementia. Being equipped with proprietary facilities and SOPs for varieties of serotypes, it provides one-stop CMO services covering AAV manufacturing, purification, and analysis. As a whole, the company leverages CARETM for both proprietary and partnered therapeutic programs with its own CMC know-how, for all-in-one preclinical development of AAV gene therapy.
Genixcure's 'XOB-031' is a recombinant AAV (rAAV) vector based on capsid-engineered AAV9, established as a lead vector from an AAV library that shows improved CNS delivery and clinical safety over wild-type AAV9. Under this MOU, Modalis will provide Genixcure with the Alzheimer’s disease treatment candidate MDL-104 based on CRISPR-GNDM® technology. Genixcure will then incorporate the GNDM-Tau gene of MDL-104 into 'XOB-031' to create a next generation AAV gene therapy for Alzheimer’s disease.
MDL-104, which uses naturally occurring AAV9 capsid as a drug delivery technology, has demonstrated significant suppression of the expression of intracellular Tau RNA and protein in target tissues and cells within the CNS upon intra-cisterna magna (ICM) administration in mouse disease models. Through a collaborative relationship under this MOU, Modalis and GENIXCURE will conduct joint research and development with the aim of transducing high levels of MDL-104 to the brain, with limited exposure to non-targeted tissues, and demonstrating long-term efficacy and safety in murine and large animal species, including NHPs, using a minimally invasive intravenous injection (IV) administration method. The combined expertise of both companies will leverage their experience in AAV engineering and epigenetic modulation to develop best-in-class, IV administered therapies for Alzheimer’s Disease and other Tauopathies.
CEO, Mr.Moon stated, “Our primary goal for this joint R&D is to verify that XOB-031 is superior to AAV9 in efficiently delivering MDL-104 to the brain via intravenous administration while minimizing exposure to non-target tissues and achieving high therapeutic gene expression.”