Translation Of RNA Medicines From Design To Clinic

RNA therapeutics comprise a rapidly expanding category of drugs that will speed clinical solutions and actualize personalized medicine. The widespread use of mRNA vaccines for COVID-19 marked 2020 as a breakout year for mRNA technology platforms.

mRNA therapeutics are applicable to cancer immunotherapies, infectious diseases, and other indications that require protein replacement therapy or antibodies. After cellular uptake, the therapeutic molecule translates genetic information into protein, an antigen, antibody, or other therapeutic protein. During development of the therapeutics several aspects must be taken into consideration–synthesis, optimization, and formulation—to deliver the cargo to the target site.

Although currently best known for their work with Pfizer on the BNT162b2 mRNA vaccine for SARS-CoV-2, according to Heinrich Haas, PhD, Vice President RNA Formulation and Drug Delivery, BioNTech, one of the company’s key areas of development is cancer immunotherapies.

To achieve this particular type of vaccination an mRNA that codes for a tumor-associated antigen (TAA) is transferred into dendritic cells, which have a key function in tumor therapy. Inside the cells the mRNA is translated and stimulates a cascade of immune responses specific to the TAA in addition to a systemic immune response.

A thorough understanding of the coherencies inside the delivery systems, such as structure and function as derived from advanced characterization, including SAXS analysis, assists performance of rational formulation development. This also helps define the critical quality attributes required to specify the product that will be important for successful translation of RNA into clinical development. 

In this article, dive deeper into R&D formulation development and clinical product development.

Reprinted with permission from GEN, published by Mary Ann Liebert, Inc., publishers; New Rochelle NY