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CellGenTech is building a pipeline of innovative gene therapies through our GMAC platform to treat patients with genetic disorders such as LCAT deficiency, hemophilia A and B, Fabry disease, and Retinitis pigmentosa.
The Basic Concept of The GMAC Platform
The basic idea of the GMAC platform is to create a novel ex-vivo gene therapy in which subcutaneous fat is collected from patients, adipocytes are isolated from the fat, target genes are transferred in adipocytes using retrovirus or lentiviral vectors, and the gene-modified adipocytes are given back into patients .
The Core Technologies Constituting the GMAC Platform
・Establishment of stable transport and preservation technology for adipose tissue.
・By utilizing the gravity of cells from the fat tissue cells in the ceiling culture system.
・Know-how to efficiently produce high-quality therapeutic cells with attention to carcinogenesis
and tumorigenesis backed by gene transfer technology suitable for adipocytes.
・Culture techniques to stabilize GMAC properties.
・Ready-to-use formulation that promotes GMAC biogenesis after GMAC administration that
can be stored for several days in a non-frozen state.
・Short period of time such as 21 days from fat collection to administration.
・Robust quality and characterization testing for the gene therapy under GCP.
GMAC Competitive Advantages
・The main advantages of GMAC over other gene therapies are higher patient safety enabling the administration in younger patients and multiple administrations, and low patient burden.
