Accelerating the transfer of research discoveries into gene therapy
Gene Therapy Medicines
Immunotherapy
Genomic Therapy
Gene therapy is a powerful medicinal approach that treats diseases by delivering specific recombinant genetic sequences to the body. In some cases, gene therapy medicines are administered in the form of "ex vivo" gene-modified cells. Gene therapy is used to treat cancer, genetic diseases, and in the future it may also treat chronic illnesses.
Immunotherapy aims to improve the body's natural immune functions to prevent or to treat diseases. Genetically-engineered lymphocytes such as CAR T cells demonstrate the vast potential of genetic immunotherapy to treat cancer. B cell genetic engineering could become a novel strategy to treat infectious diseases.
Genomic therapy describes strategies that modify the genome of somatic cells to achieve specific and stable effects. Genomic therapy can insert, delete or edit nucleotide sequences in the genome of cells and can be used to gain a new function, to inactivate or to modify gene expression. Genome editing can be very precise.
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