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Drug hunters are finding that ancient virus-like artifacts in the human genome could offer new avenues to treat neurodegeneration, cancer, autoimmunity and even aging with antibodies, vaccines and antiretroviral agents.
Although CRISPR-based gene therapy for sickle cell disease offers transformative outcomes, drugmakers are striving to develop treatments that are easy to manufacture and can reach much larger numbers of patients.
A growing number of companies are testing muscle-building agents to counter the side effects of dramatic weight loss and potentially to preserve lean muscle into old age.
Sophisticated genome editing tools to tweak multiple genes in one step are forging a first-of-its-kind generation of universal CAR T therapeutics, while ‘epitope-edited’ and virus-free manufacturing aim to democratize CAR T cells’ clinical uptake worldwide.