Rather than directly modifying the genome, this approach seeks to influence RNA expression for therapeutic benefit.
Targeting the transcriptome. Targeting the transcriptome has also become a popular approach for biotechs and attracted $230 million in VC funding in 2022. Rather than directly modifying the genome, this approach seeks to influence RNA expression for therapeutic benefit. This can bypass risks associated with permanent alterations to patients’ genetic material while still offering the ability to correct genetically based disorders. Biotechs are approaching this from diverse angles, including RNA-binding small molecules and synthetic long noncoding RNAs. Another approach in this field has been to engineer transfer RNAs that can correct mutations during transcription.