Much drug research has focused on small molecules that target disease-causing proteins. However, simple protein targets have been druggable, so some biotech companies are moving upstream by processing RNA, the molecule that carries the genetic instructions for making proteins. Johnson & Johnson sees promise in the Remix Therapeutics approach, and the pharma giant is paying $45 million to create a multi-drug research consortium.
According to the terms, this payment is an advance payment and research grant Announce Thursday. In exchange, Johnson & Johnson subsidiary Janssen Pharmaceutica received exclusive rights to three targets with applications in immunology and oncology.
Different types of RNA perform different functions. The point of Remix is to carry the type of genetic information needed to make proteins. The startup’s goal is to modulate these RNAs so that the proteins made do not cause disease. But the small molecules that Remix is developing don’t directly target RNA. Instead, Remix is designed to target the cellular components that process it.
Remix says its technology, called REMaster, identifies patterns in RNA processing and finds ways to use them to regulate RNA expression.in its website, the startup compared its approach to tuning an instrument to tune the sound. Remix’s drugs work in a variety of ways, by selectively degrading RNA to prevent protein expression, or by enhancing RNA expression to increase levels of specific proteins. Another approach is to rescue or skip damaged sections of DNA. In each case, the goal is to address the cause of the disease at its root.
RNA has been an elusive target for drug hunters because it is difficult to get small molecules to bind to it. Ribometrix and Arrakis Therapy Both use techniques that study the structure of RNA, looking for places where small-molecule drugs can bind.Ribometrix in Durham, NC has R&D Alliance with Vertex Pharmaceuticals and Genentech, a Roche subsidiary. in Waltham, MA Arrakis forms R&D partnership with Roche. Skyhawk Therapeutics is working with companies such as Bristol Myers Squibb, Merck and Takeda Pharmaceuticals to develop small molecules that target RNA.
Remix has not disclosed details about its internal pipeline.The biotech company is a relatively new player, launching in late 2020 and getting $84 million Series A financing led by Forestite Capital. Under the alliance with Janssen, Remix is eligible for milestone payments that could exceed $1 billion if any drug hits the market. The biotech will also receive royalties from the sale of any drugs commercialized by the alliance. The agreement gives Remix the option to share the clinical development of one of its programs in exchange for higher royalties.
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