Renewed interest in RNA-targeted therapies - delivery remains the achilles heel

An analysis of the recent progress in RNA therapeutics and future opportunities.

Following the COVID-19 pandemic, there has been renewed interest in developing mRNA therapeutics and vaccines. In the cell, DNA is transcribed into messenger RNA (mRNA) in the nucleus and the mRNA code is then translated to generate a protein in the cell cytoplasm. RNA-targeted therapies aim to deliver mRNA into target cells to induce the expression of specific proteins. RNA-based medicines represent a fast-growing and disruptive class of therapeutics for a breadth of disease areas.

Today, several RNA-targeted therapies are approved for commercial use, including Alnylam’s (Nasdaq: ALNY) Leqvio (inclisiran) and Isis/Genzyme’s Kynamro (mipomersen), approved for the treatment of homozygous familial hypercholesterolemia; Ionis Pharmaceuticals' (Nasdaq: IONS) Waylivra (volanesorsen) for patients with familial chylomicronaemia syndrome (FCS); Roche’s (ROG: SIX) Evrysdi (risdiplam) for the treatment of spinal muscular atrophy (SMA) and Sarepta’s (Nasdaq: SRPT) Exondys 51 (eteplirsen) for the treatment of Duchenne muscular dystrophy amenable to exon 51 skipping.