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34 Pharmaceutical Technology ® The Real Message Behind Commercial mRNA Products April eBook 2023 PharmTech.com Drug Delivery These lyophilized products can be stored for long periods at regular refrigerated temperature with- out loss of activity or significant change in physico- chemical properties. Table III denotes that key phys- iochemical properties remain unchanged after the f reeze-dr ying process. This means that ultracold storage is not required (making supply chain logis- tics much easier and cheaper). F u r t he r, t he f u nc t ion a l it y of t he R NA– c L N P c om bi n a t ion r e m a i n s l a r ge l y u n a f f e c t e d , a n d t here is retention of expression (luciferase) and/ or activity/immunogenicity (SARS-CoV-2 Spike) as illustrated in Figure 7. Figure 8 demonstrates that t he expression prof i le of lyoph i lized products is equal to 'freshly' produced and formulated products in IM formulation. Conclusion E a rl y a n d ap pr opr i a t e u s e of R NA d e s i g n a n d L N P f or mu l a t ion pl a t f or m t e c h nolo g ie s i s a n integrated way to: • Desig n R NA i n such a way to ma x i m ize t he chance of success of your therapeutic. • Produce the RNA payload at significantly high quality and in a cost-effective manner suitable for commercial-scale manufacture. • Select the delivery vehicle that corresponds to the therapeutic need. • Offer product that is stable at regular refriger- ated temperature. Careful consideration of the above points can re- sult in a better product. References 1. Rurik, J. G.; Tombácz, I.; Yadegari, A.; et al. CAR T Cells Produced In Vivo to Treat Cardiac Injury. Science. 2022, 375 (6576), 91–96. DOI:10.1126/science.abm0594 2. Wen, T.; Barham, W.; Li, Y.; et a l. NKG7 is a T-cel l-In- trinsic Therapeutic Target for Improving Antitumor C y t o t ox ic it y a n d C a nc e r I m mu n o t he r a p y. C an ce r Immunol Res. 2022, 10 (2), 162–181. DOI:10.1158/2326- 6066.CIR-21-0539 3. Eigentler, T.; et al. Phase I Study of Intratumoral CV8102 in Patients with Advanced Melanoma, Squamous Cell Carcinoma of the Skin, Squamous Cell Carcinoma of the Head and Neck, or Adenoid Cystic Carcinoma. Clinical- Trials.gov identifier: NCT03291002 4. Mauger, D. M.; Cabral, B. J.; Presnyak, V.; et al. mRNA St r u c t u r e R e g u l a t e s P r o t e i n E x pr e s s ion t h r o u g h C h a n g e s i n F u n c t i o n a l H a l f-l i f e . P r o c N a t l A c a d Sci U S A. 2019, 116 (48), 24075–24083. DOI:10.1073/ pnas.1908052116 5. Baiersdörfer, M.; Boros, G.; Muramatsu, H.; et al. A Facile Method for the Removal of dsRNA Contaminant from In-Vit ro -Tra n scr ibed m R NA. Mol T her Nucleic A cids. 2019, 15, 26–35. DOI:10.1016/j.omtn.2019.02.018. ■ A 24-hour streaming network Season 9 is streaming now! www.medicalworldnews.com For Health Care Professionals, By Health Care Professionals powered by