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Much drug development today involves developing chemicals to fit into proteins and block them. But this almost never turns out as planned: many drug candidates get broken down in the body, interfere with other proteins and cause toxic side effects, or fail to be absorbed at all.

25/8/26, 2:29 pm0 reposts3 likes

The majority of proteins are considered undruggable, because they lack a pocket for drugs to bind, or they are ‘intrinsically disordered’, without coherent structure.

What if, instead of trying to block the protein, you could simply switch it off?

siRNA, or ‘small interfering RNA’, is a short strand of RNA that blocks the production of proteins. As a brief refresher, proteins in the body are produced by DNA --> mRNA --> protein. siRNA molecules bind to the intermediate mRNA and target it for destruction, meaning the protein isn’t produced.