Poster abstracts
Poster number 55 submitted by Daniel L Kiss
The democratization of RNA therapeutics
John P. Cooke (Center for RNA Therapeutics, Houston Methodist Research Institute, Houston TX), Daniel L. Kiss (Center for RNA Therapeutics, Houston Methodist Research Institute, Houston TX), Ruli Gao (Center for RNA Therapeutics, Houston Methodist Research Institute, Houston TX), Tulsi Damase (Center for RNA Therapeutics, Houston Methodist Research Institute, Houston TX), Yi Liang, Hongye Li (Center for RNA Therapeutics, Houston Methodist Research Institute, Houston TX), Roman Sukhovershin (Center for RNA Therapeutics, Houston Methodist Research Institute, Houston TX)
Abstract:
The development of mRNA vaccines against COVID-19 brought worldwide attention to the transformative potential of RNA-based therapeutics. Integral to this potential, mRNA therapeutics are not solely the province of big biopharma. RNA therapies are a disruptive technology precisely because small biotech startups and academic researchers can rapidly develop innovative and personalized mRNA constructs. However, most of these small groups lack the key competencies to translate their transformational mRNA therapeutics into the clinic. To facilitate the democratization of mRNA therapeutics, we have built the crucial infrastructure to assist smaller groups as they bring their novel ideas to the clinic. In the Texas Medical Center, our fully integrated hospital-based RNA therapeutics program provides a single-entry point with consultation to ensure the seamless development of RNA therapy candidates into transformative drugs. Our RNA Biology and Bioinformatics faculty assist with construct design; our cGMP-trained personnel and clean rooms support the synthesis, purification, and validation of RNA drugs; plus our Nanomedicine colleagues formulate suitable lipid nanoparticles for local or systemic delivery. We have a Comparative Medicine Program with expertise in GLP preclinical studies; a first-in-human clinical trials unit for Phase 1 and 2a studies; and a large hospital system with a clinical research infrastructure that supports Phase 2 and 3 clinical trials. Furthermore, our industry partner manufactures large batches needed for Phase 2, 3 and/or commercialization. To our knowledge, we are the sole academic center with a fully integrated and operational infrastructure to support both academic groups and startups. We intend to support the translational efforts of small groups of scientists as they attain the near limitless potential of RNA Therapeutics.
Keywords: RNA therapeutics, RNA biology, RNA medicine