Breakthrough in genetic medicine: RNA gets delivered!
15 December 2022
Avidity Biosciences (in our portfolio) just released early results for their lead asset: it is a scientific feat! For the first time, RNA was specifically delivered to muscles to treat a genetic disorder. A feat opening the doors to more innovation for both patients and players in the field.
Bottom line
Targeted drug delivery, especially of a fragile drug, is a solid bottleneck for genetic medicine. In early 2021, we hypothesized that the promise of Antibody Oligonucleotide Conjugates (AOC), combining the precision of the antibody with the therapeutic effect of RNA, could be a game-changer for patients with genetic diseases. Avidity Biosciences just proved its AOC is a viable option by delivering silencing RNA to patients' muscles with minimal side effects and measurable efficacy. These results will spark the interest of investors and peers, some of whom may be willing to strike deals to benefit from such a technological breakthrough for their own genetic therapies.
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