Eighteen nucleic acid therapeutics have been approved for treatment of various diseases in
the last 25 years. Their modes of action include antisense oligonucleotides (ASOs), splice …

RNA-based gene therapy requires therapeutic RNA to function inside target cells without
eliciting unwanted immune responses. RNA can be ferried into cells using non-viral drug …

2009-05-27 Assigned to ALNYLAM PHARMACEUTICALS, INC. reassignment ALNYLAM
PHARMACEUTICALS, INC. ASSIGNMENT OF ASSIGNORS INTEREST (SEE DOCUMENT …

Delivery of genetic information to the interior of target cells in vivo has been a major
challenge facing gene therapies. This barrier is now being overcome, owing in part to …

The increasing number of approved nucleic acid therapeutics demonstrates the potential to
treat diseases by targeting their genetic blueprints in vivo. Conventional treatments …

mRNA vaccines have been demonstrated as a powerful alternative to traditional
conventional vaccines because of their high potency, safety and efficacy, capacity for rapid …

RNA-based therapeutics have shown great promise in treating a broad spectrum of
diseases through various mechanisms including knockdown of pathological genes …

Oligonucleotides can be used to modulate gene expression via a range of processes
including RNAi, target degradation by RNase H-mediated cleavage, splicing modulation …

Lipid nanoparticles (LNPs) are a potent delivery technology that have made it possible for
the recent clinical breakthroughs in mRNA therapeutics and vaccines. A key challenge to the …

Abstract mRNA vaccines have evolved from being a mere curiosity to emerging as COVID-
19 vaccine front-runners. Recent advancements in the field of RNA technology, vaccinology …