The Future of Nucleic Acid Therapies
Medicines derived from genetic building blocks have the potential to transform patient care
Brian Carothers | | Opinion
In diseases that arise from genetic mutations, most therapies address the abnormal protein that results. But not nucleic acid therapies; these revolutionary treatments target the biological pathway behind the proteins. Consider, for example, a drug that targets the mRNA upstream of protein expression, thereby affecting protein synthesis – and disease progression.
In short, nucleic acid therapies can alter protein manufacturing throughout the body – a powerful tool for fighting disease. These relatively new treatments show promising results in both the clinic and commercial markets. Market growth is extremely strong, with increasing investments in research, development, and clinical programs. Coupled with the recent uptick in regulatory approvals, the future looks bright.
These treatments are highly flexible – they not only act as gene replacements, but can also be used for immunization (with clinical trials ongoing for nucleic acid-based vaccines). What’s more, their success story actually goes further back than many people may realize. The first DNA-based therapy (formivirsen, a treatment for immunocompromised patients with cytomegalovirus retinitis) was first approved in 1998. RNA therapy is a more recent development, with the first of these (patisiran, approved for polyneuropathy caused by hereditary transthyretin-mediated amyloidosis) approved in 2018. And, though patisiran is an RNA interference therapy, other types of noncoding RNA and microRNAs are also under intense investigation as disease treatments.
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