There’s a new player in the RNA therapeutics space: WaVe Life Sciences, a Boston-Japan biotech collaboration that just closed out an $18 million Series A.
It’s working on a pipeline of stereopure nucleic acid therapeutics, which includes antisense and exon-skipping drug candidates. It says it’s working to build the “first and only” platform that can produce single, precisely defined oligonucleotide structures that have “enhanced pharmaceutical properties” compared to conventional drugs in the same class. No indications disclosed as yet.
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Fierce Biotech took a deeper dive:
The big idea at WaVe is that Verdine and Wada have constructed a new approach to developing nucleic acid drugs with a single oligonucleotide structure. In antisense programs–which currently rely on a phosphorothioate backbone containing a complex set of 524,288 isomers–the new approach would work by cleaving mRNA at specific sites; a precision chop that promises to precisely target genetic variations that trigger diseases.
They vow that this new epigenetic paradigm will be far simpler and scalable than the present-day methods in use at the leading clinical-stage companies focused on antisense, siRNA, mRNA and microRNA. And while still in preclinical development, they’re promising that this “revolutionary” new approach can avoid the potential side effects and deliverability issues that have complicated far more advanced programs.
Here’s Xconomy’s take on it:
So why does WaVe think it can make better RNA drugs? It has to do with what’s known as chirality, chemistry’s version of handedness. Just as the right and left hand have all the same components, but arranged in mirror images of one another, many drug molecules have different versions that are composed of the same atoms but in mirrored arrangements—and it turns out that handedness sometimes has a dramatic effect on the body’s response to the molecule. Thalidomide’s notorious propensity for causing birth defects is due to one version of the molecule, for instance, while its desired sedative effects are produced by the mirrored version.
WaVe’s claim—something it’ll have to prove—is that chirality is important for RNA drugs too. Bolno says that this concept has largely been “ignored” when it comes to nucleic acids and antisense drugs, but that it shouldn’t be, because it limits their potential and potency. What’s more, these molecules are larger and more complex than traditional drugs—with more places along their length for atoms to be attached in a left- or right-handed configuration. Current techniques for synthesizing these molecules don’t control for that, so they produce a mixture of thousands of different versions of a would-be drug—each with “unique pharmacology,” Bolno says.
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The round was led by RA Capital Management and Kagoshima Shinsangyo Sosei Investment.
