Ionis Antisense Programs Highlighted at the OTS Annual Meeting

SPINRAZA Lancet Publication Chosen as OTS Paper of the Year

Ionis’ Antisense Technology Platform and Programs Highlighted in 11 Presentations and Posters

Each year, the Oligonucleotide Therapeutics Society (OTS) annual meeting draws the world’s leading experts and esteemed scientists in oligonucleotide therapeutics together to discuss the latest developments in the field, including research from antisense, siRNA, immunomodulatory, aptamers, micro RNA and non-coding RNA. The goal of the society is to foster a forum for the exchange of scientific knowledge and groundbreaking new ideas, and also to recognize the outstanding work of deserving researchers via OTS annual awards. The 2017 OTS annual meeting was held in Bordeaux, France on September 24-27th.

The 2016-2017 OTS Paper of the Year was awarded to the SPINRAZA publication titled, “Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study”1.  The OTS Paper of the Year Award recognizes the most impactful paper in the field of oligonucleotide therapeutics. Ionis authors and their collaborators are delighted that the Phase 2 SPINRAZA publication was selected as this year’s recipient. Primary author Richard Finkel, M.D., chief, division of neurology, department of pediatrics, Nemours Children’s Hospital, accepted the award on behalf of the publication’s contributing authors, including Ionis’ Dr. Eugene Schneider, Dr. Daniel Norris, Shuting Xiao, Dr. Frank Bennett and Dr. Frank Rigo.

The paper highlighted positive results from the Phase 2 open-label study evaluating multiple doses of SPINRAZA in patients with infantile-onset SMA. These results spanned multiple efficacy endpoints, including event-free survival, motor function endpoints, electrophysiological measurements and developmental milestones. SPINRAZA demonstrated a favorable safety and tolerability profile in the study. Earlier this year, the interim findings of the Phase 3 ENDEAR and CHERISH studies further strengthened the results of The Lancet publication.

In addition to the Paper of the Year Award, the society highlighted ten other Ionis antisense programs and advances in its antisense technology platform, including:

  • ‘Advances in Targeted Delivery of Nucleotides Beyond the Liver’ oral presentation by Shalini Andersson, Ph.D., Senior Director Cardiovascular and Metabolic Diseases, AstraZeneca
  • ‘ATXN2 as a Therapeutic Target for SCA2 and ALS’ oral presentation by Dr. Frank Rigo, Ph.D., Executive Director of Neuroscience Drug Discovery, Ionis Pharmaceuticals
  • ‘Antisense Platform Technology: Recent Phase 3 Experience and Beyond’ oral presentation by Richard Geary, Ph.D., Senior Vice President, Antisense Drug Development, Ionis Pharmaceuticals
  • ‘ASO-mediated Modulation of Readthrough and the Nonsense Mediated Decay Pathway to Upregulate W1282X CFTR’ oral presentation by Melissa Keenan, Ph.D., Postdoctoral Scientist in Antisense Drug Discovery, Ionis Pharmaceuticals
  • ‘Nusinersen Treatment of Spinal Muscular Atrophy’ oral presentation by Richard Finkel, M.D., Chief, Division of Neurology, Department of Pediatrics, Nemours Children’s Hospital
  • ‘Preclinical efficacy of SOD1 antisense oligonucleotide in phase 1 testing for SOD1 amyotrophic lateral sclerosis’ poster presentation by Alexander McCampbell, Ph.D., Principal Investigator, Biogen
  • ‘Experimental and model-based characterization of antisense oligonucleotide (ASO) distribution in the cerebral spinal fluid (CSF)’ poster presentation by Michael Monine, Ph.D., Senior Pharmacometrician, Biogen
  • ‘Antisense Oligonucleotide-mediated Upregulation of CFTR via Steric Block of Post-transcriptional Control Elements: A Novel Approach for Cystic Fibrosis Therapeutics’ poster presentation by Shruti Sasaki, Ph.D., Postdoctoral Fellow, Ionis Pharmaceuticals
  • ‘Cellular Fractionation and Evaluation of Hepatic Cells after Antisense Oligonucleotide Treatment in Mice’ poster presentation by Sue Murray, Director of Antisense Drug Discovery, Ionis Pharmaceuticals
  • ‘Generation 2.5 Constrained Ethyl Antisense Oligonucleotide Renal Sub-Organ Pharmacodynamics in the Healthy Rodent Kidney’ poster presentation by Emily Brand, Senior Research Associate, Ionis Pharmaceuticals

Complete abstracts for Dr. Richard Finkel’s Paper of the Year Award presentation and others can be accessed on the OTS website at


  1. Finkel et al., Lancet, Advanced Online Publication, December 6, 2016 (link: