AstraZeneca and Isis Pharmaceuticals Develop Novel Oligonucleotide Delivery Methods

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The new partnership will focus on RNA biology for the discovery and manufacture of new therapies.

AstraZeneca and Isis Pharmaceuticals announced on Nov. 13, 2013 that the companies plan to collaborate on the discovery of antisense oligonucleotides, which are short, single-stranded DNA or RNA molecules. These small pieces of nucleic acids are being investigated to treat genetic disorders. Rather than working on specific proteins like many other pharmaceuticals, antisense therapies work by inhibiting the translation of messenger RNA, effectively blocking the function of the genetic material that is thought to be causative of disease.

Antisense therapies are challenging to produce, as some antisense compounds fail to enter cells they are meant to target. The new delivery methods, which draw on Isis Pharmaceuticals’ Ligand Conjugation Antisense (LICA) technology, aim to facilitate access of antisense oligonucleotides into specific organs and cells. The initial disease state focus areas for the collaboration will be oncology and cardiovascular and metabolic conditions.

The collaboration builds on an existing relationship with Isis Pharmaceuticals and AstraZeneca. Together, the two companies already have two antisense compounds in development: AZD9150, a first-in-class, antisense oligonucleotide inhibitor of STAT3, and AZD5312, an antisense oncology compound.

"This exciting collaboration very much supports AstraZeneca's research and development in the area of RNA-based therapeutics,” said Susan Galbraith, head of the oncology innovative medicines unit at AstraZeneca, in a press release. “If successful, we'll have a way to selectivity modulate therapeutic targets in specific cell types that are intractable to small molecules and antibodies.”


Isis Pharmaceuticals already has experience harnessing antisense technologies for drug development and delivery. The company’s orphan drug Kynamro (mipomersen sodium), which treats homozygous familial hypercholesterolemia, a condition that causes extremely high cholesterol levels and heart attacks at an early age, is an antisense therapy. That drug was marketed by Sanofi’s Genzyme division and was approved by FDA in January 2013.

Source: Isis Pharmaceuticals