RNA molecules play a variety of important roles in cells, including structural (ribosomal RNA), enzymatic (small nuclear RNA), and information transfer (messenger RNA). The relatively large size and well-known instability of RNA would make it an unlikely vehicle for drug development but, now, technological advances have helped overcome those issues.
Indeed, mRNA technology allowed Moderna and Pifizer-BioNTech to shorten the time to market of their COVID-19 vaccines dramatically compared with traditional vaccine development cycles.
A session at the DIA Annual Meeting 2021 on Monday June 28 at 4pm EDT will be discussing different examples of RNA based therapeutics, as well as some regulatory considerations.
The speakers include Philip (P.J.) Brooks, program director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS), US NIH, Girish Chopda, associate Director, nonclinical development, Dicerna Pharmaceuticals, Christopher Saeui, biologist, Pharm/Tox/OTAT, CBER, US FDA, and Philip Santangelo, professor, Coulter Department of Biomedical Engineering, Georgia Institute of Technology and Emory University School of Medicine.
A recently published study in Frontiers in Bioengineering and Biotechnology examines the challenges and advantages associated with use of RNA-based drugs along with various approaches for RNA delivery.
Disruptive technology
Many in the industry believe the mRNA approach should accelerate new vaccine and drug development timetables and serve as a disruptive technology for the wider pharmaceuticals space.
Pfizer and Moderna are optimistic they will be able to use their messenger RNA technology to combat other diseases and illnesses.
Moderna is deploying mRNA for use in new vaccines that hopefully will provide immunity against diseases such as respiratory syncytial virus (RSV), cytomegalovirus (CMV), human immunodeficiency virus (HIV), Zika, Epstein-Barr Virus, and influenza, among others. “Even as we have shown that our mRNA-based vaccine can prevent COVID-19, this has encouraged us to pursue more-ambitious development programs within our prophylactic vaccines modality,” Stéphane Bancel, Moderna’s chief executive officer
Pfizer CEO Albert Bourla, speaking at Bio Digital 2021 this week, said that, in terms of a technology, mRNA has proven to be "very powerful" and Pfizer and others have only "scratched the surface" of what can be achieved with this tool.
Sanofi gained an mRNA immunotherapy platform with its acquisition of Tidal Therapeutics in April.
The Cambridge, Massachusetts biotech has a novel mRNA-based approach for in vivo reprogramming of immune cells. The technology is based on proprietary nanoparticles that deliver mRNA to selectively targeted immune cells in the body and reprogram them.
The in vivo approach is designed to provide similar efficacy to current ex vivo approaches (where immune cells are genetically modified to enhance their therapeutic properties - such as chimeric antigen receptor CAR-expressing T-cells). However, an in vivo approach offers the potential for improved safety, outpatient dosing, and repeat dosing, according to the companies.
Sanofi will use the platform to expand its research capabilities in immuno-oncology and inflammatory diseases, while noting that it is likely to have “broad applicability” to other disease areas as well.