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WuXi Xpress White Paper: RNA therapeutics to become a dominant treatment modality within a decade

Industry experts speaking within a newly released WuXi Xpress White Paper on RNA have identified 2019 as potentially a key turning point for the modality, sparking the beginnings of a new era for RNA therapeutics.

After more than two decades of research, with Onpattro’s approval by both the US FDA and EU in 2018, the white paper suggests we may now see increasing numbers of therapies brought to patients. Especially, as many advances have been made in the last decade to make the delivery of RNAs more stable. In fact, more than 69 companies are now developing RNA therapeutics across some 315 clinical trials.

“Initially, DNA-based approaches were favored in drug development based on the inadequate delivery technologies for the less stable RNA. However, over the last ten years the field has seen major advances and RNA therapeutics offer a more flexible approach to address a variety of diseases compared to DNA-based therapeutics,” said MiNA Therapeutics CEO, Robert Habib, an expert contributor to the White Paper.

RNA therapeutics offer advantages as they enter the cell’s cytoplasm ready to take-over its protein manufacturing machinery, whereas DNA therapeutics, such as gene therapy and gene editing technologies, must penetrate the cell’s nucleus to modulate genes and risk altering the genome.

The report discusses the vast potential RNA therapeutics could have due to the variety of different mechanisms they use to combat disease – resulting in the up- or down-regulation of certain proteins in a cell. RNA therapeutics include antisense and RNAi – which are short interfering RNA (siRNA) and microRNA (miRNA) – messenger RNA (mRNA), self-amplifying mRNA (samMRNA) and small activating RNA (saRNA), as well as the alternative strategy of using small molecules as modulators of RNA. 

Emphasising the impact they could have over the next decade, Arrakis CEO, Michael Gilman, Ph.D. observed “85% of the human genome is transcribed into RNA” and only 3% translates into proteins, many of which cannot be targeted by existing treatment modalities.

“So, between these undruggable proteins and all the emerging biology of noncoding RNAs, there is a lot of disease biology that remains beyond our reach,” Gilman explained. “By directly targeting RNA, we can bring that biology into play for patients.”

To explore the potential and scope of RNA therapeutics, WuXi Xpress spoke with Habib, Gilman and the leaders of four other companies developing medicines targeting RNA: Keith Blundy, Ph.D., CEO of STORM Therapeutics; Karin Jooss, Ph.D., executive vice president and chief scientific officer of Gritstone Oncology; Patrick Lu, Ph.D., founder, president and CEO of Sirnaomics; and Roel Schaapveld, CEO of InteRNA Technologies.

To read the complete white paper or the full transcripts of the interviews, please visit WuXi Xpress.

STORM Therapeutics is using RNA-modifying enzymes to develop first-in-class drugs in oncology and other diseases. InteRNA develops miRNA drug candidates specializing in cancer. Sirnaomics uses proprietary Polypeptide Nano-Particle (PNP) technology for small interfering RNA (siRNA) drug delivery targeting the tumor micro-environment (TME), as well as various cell types in the liver. Arrakis is working on small molecules that directly bind to RNA involved in protein expression. Gritstone uses self-amplifying mRNA (samRNA) encapsulated in a Lipid Nanoparticle (LNP, a synthetic delivery system) to develop neoantigen-based immunotherapies. MiNA Therapeutics is exploring saRNAs (small activating RNAs) for advanced liver cancer.

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Last Updated: 30-Jul-2019