In August of 2018 the Food and Drug Administration (FDA) approved the first of a potentially game-changing class of drugs called small interfering ribonucleic acid (siRNA). FDA Commissioner Scott Gottlieb, MD announced: “New technologies like RNA inhibitors that alter the genetic drivers of a disease, have the potential to transform medicine so we can better confront and even cure debilitating illnesses.”
This new class of drugs, which is a direct result of the Human Genome project, is unique because the drugs work at the RNA level to specifically silence the production of a disease-causing protein. RNA therapies can be designed to interfere with the production of abnormal proteins throughout the body. Initially focused on infectious diseases and oncology, this technology is now being applied across many therapeutic areas. Whereas DNA technologies – such as gene therapy or gene editing try to fix a dysfunctional gene or re-introduce an intact version of a gene – these new RNA therapies have the ability to utilize a cell’s own internal workings to alter the expression of genes. This gives RNA technology an advantage over DNA technologies because of their wider applications.
RNA technology has the potential to get at the root causes of disease and help bring novel drugs to patients. In this WuXi AppTec Innovator series we will explore the RNA pioneer researchers and companies. STORM Therapeutics is one of the earliest of these trailblazing companies, founded in 2015, as a spinout of University of Cambridge. The company built on the ground-breaking RNA epigenetics work of Professors Tony Kouzarides and Eric Miska for the discovery of first-in-class drugs in oncology and other diseases. It is leading the industry in tackling disease through modulating RNA-modifying enzymes and is developing a unique platform to address these, including RNA methyltransferases. STORM is backed by blue-chip investors Cambridge Innovation Capital, M Ventures, Pfizer Ventures, IP Group and Taiho Ventures LLC.
At the helm of STORM is Keith Blundy, PhD, Director and Chief Executive Officer. Keith joined STORM Therapeutics as CEO in January 2017. Prior to STORM, Keith was Chief Executive of Cancer Research Technology Ltd (CRT), the commercial arm of Cancer Research UK (CRUK), where he drove the development of CRT to become a global leader in the commercialisation of cancer discoveries. He was instrumental in the launch of the £70m Cancer Research Technology Pioneer Fund in partnership with the European Investment Fund (EIF) and BACIT, developed risk sharing drug discovery alliances with AstraZeneca, Teva (formerly Cephalon), Forma, Merck, and MRCT.
Follow link to read Keith Blundy’s insightful interview with Wuxi App Tec