Abstract
The hundreds of individual muscles in the human body have long vexed drug developers trying to devise therapies that restore strength to people with genetic muscle diseases. Oligonucleotides designed to patch or block the broken genes that characterize these conditions don’t do a very good job of navigating to muscles on their own. That’s led drug companies to douse patients with high concentrations of oligonucleotide therapies, hoping that enough of the molecules will make their way into muscle. The results have been marginal. To solve that problem, a start-up called Dyne Therapeutics has raised $50 million in series A financing to develop antibody-oligo conjugates that shuttle oligo therapies into muscle cells. The idea takes a play from the antibody-drug conjugate field, where toxic anticancer drugs are attached to tumor-homing antibodies to ensure that the drugs exert their potent effects solely on cancer cells. “When you deliver a naked oligo, very
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