Over the past several years, small biotech led significant progress in the development and clinical validation of nucleic acid therapeutics (NATs) across a range of therapeutic areas, largely for rare disorders, such as: Ionis’ Kynamro (homozygous familial hypercholesterolemia), Sarepta’s Exondys 51/Vyondys 53 (Duchenne muscular dystrophy), Biogen’s Spinraza (spinal muscular atrophy), Alnylam’s Onpattro (hTTR amyloidosis polyneuropathy) and Givlaari (acute hepatic porphyria). During this time, Large Biopharma, bet on specific NAT platforms: antisense oligonucleotides (ASOs), a variety of RNA Inhibition (RNAi) technologies and messenger RNA (mRNA) augmentation, but Pharma’s recent intense interest is driven by validation within broader diseases like hepatitis B and hypercholesterolemia and secondary cardioprevention, having met endpoints in pivotal trials. In response to these clinical wins, and with the anticipation of additional approvals on the horizon, there has been a flurry of deal activity (Billion plus dollar deals each: Alnylam/Regeneron, Arrowhead/J&J, Dicerna/Roche and Novo Nordisk) and developers with NATs in their portfolios have generally seen an increase in shareholder value and lucrative exits with the watershed $9.7 billion acquisition of The Medicine’s Company by Novartis for inclisiran, a PCSK9 GalNAc conjugated siRNA licensed from Alnylam. However, beyond these successes, challenges remain as a barrier to developing and commercializing NAT drugs, such as: improving therapeutic index across novel targets and diseases, many of which, delivery to affected tissues is insufficient.
Join Cello Health BioConsulting and our distinguished panel of nucleic acid therapeutic experts as we discuss the technology behind these innovative platforms, how to navigate the remaining scientific, clinical & commercial challenges, and what upcoming value-inflecting events can be expected.