mRNA & RNA Therapeutics

mRNA platforms have moved well beyond COVID into cancer, rare disease and in vivo cell therapy. The session covers personalised cancer mRNA vaccines (BNT122, mRNA-4157 KEYNOTE-942 in melanoma), in vivo CAR-T using mRNA-LNP delivery (Capstan, BioNTech BNT211), protein-replacement mRNA for rare diseases (PKU, methylmalonic acidemia), siRNA therapeutics (inclisiran, vutrisiran), antisense oligonucleotides (tofersen, ulefnersen), and the manufacturing scale-up. Discussion addresses self-amplifying RNA, the comparison of LNP vs other delivery, RNA-editing therapeutics, the regulatory pathway for personalised mRNA, and emerging cardiovascular indications.