Expanding the RNAcore to Encompass the Entire Cycle of a Cure

Expanding the RNAcore to Encompass the Entire Cycle of Translation

The RNAcore began as a core group for the National Heart, Lung and Blood Institute Progenitor Cell Biology Consortium, producing high-fidelity research and clinical-grade RNA, including mRNA, modified mRNA and noncoding RNA, for the support of fundamental research and clinical applications.

In 2015, the Houston Methodist RNAcore was awarded $4.8 million from the Cancer Prevention and Research Institute of Texas (CPRIT) to expand the small RNAcore supporting basic research and RNA construct development. At the time, the RNAcore was the first academic entity in Texas to generate the new class of drugs known as RNA therapeutics. The grant funded support of the development and generation of RNA therapeutics, particularly for cancer immunology. Fast-forward six years, and RNA therapeutics are on the tip of everyone’s tongue. The development of mRNA vaccines against COVID-19 brought worldwide attention to the transformative potential of RNA-based therapeutics.

In 2020, CPRIT awarded Houston Methodist with an additional $4 million to expand the RNAcore into a state-of-the-art comprehensive RNA therapeutics facility that further enables academic and biotechnology groups to translate their ideas and innovations into therapies by providing services in

development, manufacturing, quality control and preclinical and early-stage clinical testing of RNA drugs. The RNAcore’s industry partner VGXI has licensed their manufacturing processes to scale-up for late-stage trials and commercialization. The CPRIT funding helps the RNAcore to accelerate the drug discovery process for RNA therapeutics by significantly reducing the time needed to bring potential new treatments from bench to bedside.

Under the leadership of John P. Cooke, MD, PhD, Joseph C. “Rusty” Walter and Carole Walter Looke Presidential Distinguished Chair in Cardiovascular Disease Research, and principal investigator of the CPRIT grant, the RNAcore has supported the development of several RNA-based therapies, including an mRNA vaccine for metastatic melanoma cancer treatment. Promising preclinical data showing dose-dependent antitumor activity with the vaccine led to the creation of a spin-off company focused on mRNAbased vaccine products for cancer. Daniel L. Kiss, PhD, assistant professor of cardiovascular sciences, working with the RNAcore, has generated circular RNAs to block the activity of overexpressed microRNAs in breast cancer. There are many biotech companies and academic institutes that can quickly develop these therapies, but most lack the means to translate their product into the clinic. Researchers at Houston Methodist have built critical infrastructure to support the democratization of mRNA therapeutics. This program is a single-entry point for the development of RNA therapy candidates into transformative drugs and might be the only program of this sort in an academic center.

Though the concept of using RNA-targeting for therapeutic purposes has been around for a while, clinical potential is only just being realized, an example being the COVID-19 mRNA vaccines. With CPRIT funding, the RNAcore will help accelerate the drug discovery process for RNA therapeutics by significantly reducing the time needed to bring potential new treatments from bench to bedside. ”

In 2020, under the leadership of John P. Cooke, MD, PhD, and principal investigator of the CPRIT grant, CPRIT awarded Houston Methodist with an additional $4 million to expand the RNAcore into a state-of-the-art comprehensive RNA therapeutics facility. – John P. Cooke, MD, PhD

Joseph C. “Rusty” Walter and Carole Walter Looke

Presidential Distinguished Chair in Cardiovascular Disease Research

Chair, Department of Cardiovascular Sciences

Professor of Cardiovascular Sciences

Houston Methodist