AstraZeneca to Partner with Isis Pharmaceuticals to Advance Antisense Oligonucleotides Used in SMA Therapy

Isis Pharmaceuticals, Inc. and AstraZeneca announced a new partnership to develop new, more efficient delivery methods of its antisense oligonucleotides to the desired tissue. Isis is currently utilizing its antisense technology to develop ISIS-SMNRx, an experimental therapy for spinal muscular atrophy being developed in collaboration with Biogen Idec. This new collaboration between AstraZeneca and Isis Pharmaceuticals’ research will seek to continue to advance the field of antisense oligonucleotide-based therapeutics and RNA biology, which will target a wide range of diseases, including cancer, cardiovascular, and metabolic diseases.

Antisense oligonucleotides represent a new exciting field in molecular therapeutics, because they can fine tune the expression of genes by regulating messenger RNAs, thus, before the production of proteins. For SMA, the Antisense oligonucleotide ISIS-SMNRx works by repairing the genetic defect in the disease that leads to its deadly, progressive destruction of muscle. The new methods will be designed with Isis Pharmaceuticals’ successful Ligand Conjugation Antisense (LICA) technology to allow for a better access of Antisense oligonucleotides to specific organs. An improved specificity allows for a reduction in the therapeutic dose. This is highlighted by a previous result obtained with Isis’ GalNac-conjugated antisense oligonucleotides that exhibits better liver target delivery which allowed a reduction of approximately 10-fold in the dosage.

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Susan Galbraith, Head of the Oncology Innovative Medicines Unit, AstraZeneca commented, “This exciting collaboration very much supports AstraZeneca’s research and development in the area of RNA-based therapeutics. If successful, we’ll have a way to selectivity modulate therapeutic targets in specific cell types that are intractable to small molecules and antibodies. This could lead to a number of ground-breaking drugs for both oncology and cardiovascular and metabolic diseases.”

Brett Monia, Senior Vice President of Antisense Drug Discovery, Isis Pharmaceuticals added, “The collaboration expansion announced today builds upon an already successful agreement between Isis and AstraZeneca. Together, we have advanced ISIS-STAT3Rx and ISIS-ARRx in clinical development, both of which are being evaluated in patients with cancer. This opportunity also complements our internal efforts to expand the use of our technology and develop drugs with broad therapeutic applicability.”

Marcus Schindler, Head of the CVMD Innovative Medicines Unit, AstraZeneca finally noted, “RNA molecules play an increasingly important role in our research portfolio. We are delighted to be expanding our existing, strong collaboration with Isis Pharmaceuticals, who are leading players in RNA biology, with the aim of improving the delivery of antisense oligonucleotides to specific cardiovascular and metabolic tissue targets.”