From Cancer Progress 2022

Moderator: Michael C. Rice, MS, MBA, SVP, BioConsulting, Head of Advanced Therapeutics and Rare Diseases, Lumanity

Panelists:

  • Jacob Becraft, PhD, CEO, Co-founder, Strand Therapeutics
  • Dietrich A. Stephan, PhD, Chairman and CEO, NeuBase Therapeutics
  • Maria Luisa Pineda, PhD, Chief Executive Officer and Co-Founder, Envisagenics, Inc.
  • Leslie Williams, Co-Founder, President & CEO, hC Bioscience, Inc.

The last serval years have demonstrated the versatility and adaptability of therapeutic platforms that target RNA or where RNA is the therapeutic payload. These nucleic acids therapeutics are biologically tractable and can be designed by simply typing in the complementary sequence of a target gene or mRNA of interest into a mechanical synthesizer that can manufacture pharmaceutical grade oligonucleotides, or biologically express RNA to clinical scale. 

However, delivery to the affected tissues has been the major barrier to achieving clinical therapeutic potential for as unmodified RNA is short-lived due to nuclease degradation and targeting an affected cell, having that cell take up the therapeutic and release the RNA into its cytoplasm has plagued the field. To overcome delivery barriers, chemical modifications, GalNAc receptor targeted conjugates and Lipid Nanoparticles (LNPs), have facilitated antisense and siRNA therapies to access to vital organs such as the liver and enabled the rapid development and emergency use authorization of a new class of vaccines for the COVID19 pandemic (BioNTech/Pfizer and Moderna). These successes have not only accelerated the advancement of nucleic acid chemistries, formulation, manufacturing and enabling delivery technologies but also created >$130B in revenue in product sales over the past two years. 

Now that these BioPharma firms are cash-rich and in need of new therapeutic opportunities beyond infectious disease and genetic liver diseases, they see oncology as an attractive adjacency. The goal is to open of oncology opportunities by addressing undruggable oncogenes and tumor driver mutations, induce apoptosis and potentiate the antitumor immune response.  Today’s panel will discuss recent advances on both the RNA therapeutic payloads and enabling delivery technologies and potential as anticancer strategies.