Pharma’s roller-coaster relationship with RNA therapies

  title={Pharma’s roller-coaster relationship with RNA therapies},
  author={Michael Eisenstein},
After a groundswell of hype and a sceptical backlash, the pharmaceutical industry is learning how to leverage RNA interference in the clinic. After a groundswell of hype and a sceptical backlash, the pharmaceutical industry is learning how to leverage RNA interference in the clinic. 

Paving the Road for RNA Therapeutics

Chemoenzymatic Preparation of Functional Click‐Labeled Messenger RNA

Modular and simple methods to chemoenzymatically modify mRNA are described and it is demonstrated that the modified mRNA is accepted by the translation machinery.

The effect of miR-101 on the memory of rats with a model of Alzheimer’s disease

Intranasal administration of liposomal miRNA miR-101 involved in liposomes to rats with a model of early manifestations of Alzheimer’s disease improved neuroethological parameters of working and spatial memory and restored the level of research activity and differentiation of familiar and unfamiliar objects in the testing of working memory in rats.

Evolution of complexity in non-viral oligonucleotide delivery systems: from gymnotic delivery through bioconjugates to biomimetic nanoparticles

This review will present the evolution of complexity of oligonucleotide delivery methods with focus on increasing complexity of formulations from gymnotic delivery to bioconjugates and to lipid nanoparticles in respect to developments that will enable application of therapeutic oligon nucleotides as drugs in personalized therapies.



Safety and efficacy of RNAi therapy for transthyretin amyloidosis.

BACKGROUND Transthyretin amyloidosis is caused by the deposition of hepatocyte-derived transthyretin amyloid in peripheral nerves and the heart. A therapeutic approach mediated by RNA interference

Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles

Evidence is provided of inducing an RNAi mechanism of action in a human from the delivered siRNA and the presence of an mRNA fragment that demonstrates that siRNA-mediated mRNA cleavage occurs specifically at the site predicted for anRNAi mechanism from a patient who received the highest dose of the nanoparticles.

Gene expression: RNA interference in adult mice

It is shown that transgene expression can be suppressed in adult mice by synthetic small interfering RNAs and by small-hairpin RNAs transcribed in vivo from DNA templates.

Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans

To their surprise, it was found that double-stranded RNA was substantially more effective at producing interference than was either strand individually, arguing against stochiometric interference with endogenous mRNA and suggesting that there could be a catalytic or amplification component in the interference process.