Research in preclinical nucleic acid therapeutics is exploring various models and approaches for developing new treatments. These efforts encompass a range of diseases, including cancer, rare neurodevelopmental disorders, obesity, and demyelinating diseases, with some studies focusing on specific therapeutic classes like circular RNA and antisense oligonucleotides. The development of preclinical models, including the use of AI-driven virtual cell models, is being evaluated to improve the translation of research into effective medicines.