Nav: Home

Thirty years of innovation pays off as oligonucleotide therapeutics come to market

January 25, 2018

The recent approval of SpinrazaTM (nusinersen), jointly developed by Ionis Pharmaceuticals and Biogen, marks the arrival of a new class of biological products - oligonucleotide therapeutics. A recent publication from the Center for Integration of Science and Industry at Bentley University shows that the thirty year path from the initiation of research on oligonucleotides as therapeutics to the emergence of effective products followed predictable patterns of innovation, in which novel products are successfully developed only after the underlying basic research reaches a requisite level of maturity.

The article in the journal Molecular Therapy - Nucleic Acids titled "As Technologies for Nucleotide Therapeutics Mature, Products Emerge" describes the analysis of more than 60,000 research publications related to oligonucleotide therapies. The work applies a data analysis model for the growth and maturation of biomedical research to characterize the progress of research on the pharmaceutical chemistry of oligonucleotide therapeutics, as well as the molecular targets, for their actions. Previous work has demonstrated that successful product development characteristically follows the maturation of the enabling research past an analytically defined established point. The present work shows that the approval of oligonucleotide therapeutics exhibits a similar pattern.

Oligonucleotide therapeutics are a novel class of biopharmaceutical products comprised of short strings of synthetic nucleotides, which resemble the building blocks for DNA. These products are designed to alter the expression of target genes in the body, suppressing expression of genes associated with disease, or restoring expression of genes required for health. Originally described in the 1980s, the potential of oligonucleotides as therapeutic agents spawned hundreds of clinical trials and billions of dollars of investment in companies developing these therapeutics without generating successful products.

"Discovery and development of new medicines, such as an oligonucleotides, requires extensive basic research to optimize the product chemistry, improve its biological activity, and characterize a specific chemical entity that can safely treat diseases," said Dr. Fred Ledley, director of the Center for Integration of Science and Industry. "Our work suggests that when data analytics are used to track the maturation of basic research, drug developers will be able to more efficiently recognize the point at which the science is mature enough for product development."
-end-
The work was supported by a grant from the National Biomedical Research Foundation.

THE CENTER FOR INTEGRATION OF SCIENCE AND INDUSTRY at Bentley University brings together faculty, students, and visiting scholars in an interdisciplinary effort to understand and accelerate the translation of scientific discoveries to create public value. The Center is an environment for thought leadership and interdisciplinary scholarship spanning basic science, data analytics, business and public policy. For more information, visit http://www.bentley.edu/sciindustry and follow us on Twitter @sciindustry.



Bentley University

Related Innovation Articles:

Shaping the future of health innovation
Future advances in healthcare will be aided by a new £10 million facility -- the National Institute for Health Research Innovation Observatory based at Newcastle University, UK.
Building on the foundations of innovation
The new issue of Technology and Innovation has a special section on the 2016 NAI Conference, including articles on gender and bias in science, the history of the National Academy of Inventors, alternative rubber crops, and the next industrial revolution.
What is innovation, and how can we awaken its dormant traits and cultivate them?
While education may not be able to create innovative traits in individuals, education may improve the ability of individuals to utilize the traits they already possess.
LA BioMed's 4th Annual Innovation Showcase
The Innovation Showcase will be attended by over 300 entrepreneurs, investors, executives from biotech and pharma medical devices companies, legal experts, service providers, prominent scientists, and technology transfer personnel from premier academic institutions.
Accelerating low-carbon innovation through policy
Will innovative technologies contribute to mitigating climate change? Learn about the successes and failures of low-carbon technology and how policy instruments help and hinder technological innovation.
The next Horizon: Strategies to promote European competitiveness in innovation
EU-LIFE, the alliance of research centers in life sciences to support and strengthen European research excellence released today a statement with its recommendations for next framework program for Research & Innovation, FP9.
Can we put a price on healthcare innovation in cancer?
Is there evidence that the money spent on innovation 'for the cure' actually benefits cancer patients?
Alzheimer's treatment innovation pipeline is building
A new analysis of the Phase II Alzheimer's drug pipeline, conducted by ResearchersAgainstAlzheimer's, revealed 57 new Alzheimer's drugs.
Business investment in innovation pays off
Firms need to invest in innovation in order to create new technologies and move the economy forward, according to new research from the University of Houston.
Technology and innovation not driven by climate change
Middle Stone Age of southern Africa is a period of dramatic innovation in subsistence, cultural and technical practices.

Related Innovation Reading:

Best Science Podcasts 2019

We have hand picked the best science podcasts for 2019. Sit back and enjoy new science podcasts updated daily from your favorite science news services and scientists.
Now Playing: TED Radio Hour

Bias And Perception
How does bias distort our thinking, our listening, our beliefs... and even our search results? How can we fight it? This hour, TED speakers explore ideas about the unconscious biases that shape us. Guests include writer and broadcaster Yassmin Abdel-Magied, climatologist J. Marshall Shepherd, journalist Andreas Ekström, and experimental psychologist Tony Salvador.
Now Playing: Science for the People

#513 Dinosaur Tails
This week: dinosaurs! We're discussing dinosaur tails, bipedalism, paleontology public outreach, dinosaur MOOCs, and other neat dinosaur related things with Dr. Scott Persons from the University of Alberta, who is also the author of the book "Dinosaurs of the Alberta Badlands".