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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.
Our ranking is out, and it has a few surprises. In pole position — predictably — is Wegovy, the weight-slashing GLP-1 agonist, with AI-hallucinated proteins a close second. These are followed by drugging options for RNA. The dark horses are virus-free gene therapies and a sweet protein.
The go-ahead for Vertex’s gene editing therapy in sickle cell disease and β-thalassemia is a historic milestone, but this one-time treatment is costly.
A new era of brain-penetrant therapies is opening up, as companies hook up drugs to transferrin receptors or use viral capsids to hitch a ride into previously inaccessible regions.
Drugs commonly used in rheumatology, the JAK inhibitors, are opening up treatment options for people with autoimmune-driven hair loss, and new mechanistic knowledge is also helping scientists tackle androgenic alopecia, the commonest cause of baldness.