【YSA x Z-Park】Frontiers in RNA Therapy

What is RNA?

RNA, the abbreviation for ribonucleic acid, serves as the intermediary between genes and the proteins that they code for.

What is mRNA therapy?

Messenger ribonucleuc acid, or mRNA for short, plays a vital role in human biology, specifically in a process known as protein synthesis. mRNA is a single-stranded molecule that carries genetic code from DNA in a cell’s nucleus to ribosomes, the cell’s protein-making machinery.

mRNA is the set of instructions by which cells make all proteins and send them to various parts of the body. Using mRNA as a medicine is a fundamentally different approach than treating disease with other drug classes. mRNA medicines take advantage of normal biological processes to express proteins and create a desired therapeutic effect. This enables the potential treatment of a broad spectrum of diseases, many of which cannot be addressed with current technologies. mRNA has the potential to transform how medicines are discovered, developed and manufactured.

mRNA medicines aren’t small molecules, like traditional pharmaceuticals. And they aren’t traditional biologics (recombinant proteins and monoclonal antibodies) – which were the genesis of the biotech industry. Instead, mRNA medicines are sets of instructions. And these instructions direct cells in the body to make proteins to prevent or fight disease.

What is RNA silencing (RNA inhibition, or RNAi)?

A newer method that also prevents a particular gene from making protein is called RNA inhibition (RNAi). Again, the aim is to silence the gene by targeting RNA, which carries the genetic message to the protein assembly plant in the cytoplasm. By accident, scientists working in the 1990s to enhance the purple color in petunias discovered that a short piece of double-stranded RNA will cause destruction of a corresponding gene. This process is probably part of a cell’s defenses against viruses, as many viruses are paired strands of RNA. Mammalian cells usually have only single stranded RNA. Researchers now seek to design short RNAs (called short interfering RNAs, or siRNAs) that will prompt a cell to destroy matching RNA, preventing the production of a protein from a particular gene.

Speaker

• Huijuan Li, Ph.D. | Head, Analytical Development | Moderna Therapeutics
• Dr. Huijuan Li is currently the Head of Analytical Development (Executive Director) within Technology Development at Moderna Therapeutics.
• Prior to join Moderna, Dr. Huijuan Li was the director of analytical development within Biologics Discovery (GlycoFi site) and later in Bioprocess Development, Merck Research Laboratory. Huijuan and her team have successfully supported the development, regulatory filing and commercialization of two monoclonal antibodies, KEYTRUDA® and ZINPLAVATM. Huijuan also worked at Biotechnology Development at BMS in supporting the manufacturing of ORENCIA®.
• Huijuan received her PhD from University of Melbourne, Australia in 1998. From there she pursued her postdoctoral studies at Biology Department, McGill University, Montreal, Canada.
• Karyn Schmidt, Ph.D. | Scientist | Alnylam Pharmaceuticals
• Dr. Schmidt received her B.S and Ph.D. from University of Rochester. She finished her Post-doctoral Fellow at Dana-Farber Cancer Institute, Harvard Medical School. Currently, she is working as Scientist at Alnylam Pharmaceuticals.