A Momentous CRISPR Milestone was Achieved with Remarkable Results
Intellia Therapeutics and Regeneron Pharmaceuticals’ recent announcement is a major milestone worth celebrating. They shared interim data from the first ever human study using systemically administered CRISPR as a gene editing therapy. Early results that surpassed expectations show that not only is the CRISPR-based ...
OTS President’s Paper Pick– July 2021
By: Annemieke Aartsma-Rus, Ph.D. Safety, Immunogenicity, and Efficacy of the BNT162b2 Covid-19 Vaccine in Adolescents. Frenck et al. N Engl J Med. 2021 May 27; DOI: 10.1056/NEJMoa2107456 While waiting in line to receive my second Moderna Covid-19 mRNA vaccine, I read the recent paper on ...
Oligonucleotide Therapeutics – Restoring Sight to the Blind
Vision is one of our most priceless senses. Without it, we miss out on precious visuals, such as witnessing a child’s face light up with joy or seeing a glorious sunset, and even the important relational activity of reading the expressions on someone’s face ...
Can Oligonucleotides Target RNA Splicing to Treat Disease?
The human body is phenomenally complex, and we are continually discovering incredible facts about it. One fascinating aspect lies in splicing of RNA as it is processed in the cell. Alternate splicing of a single pre-mRNA molecule can create tens of thousands of different ...
How to test for immune activation by your therapeutic oligonucleotide
Presenter: Arthur M. Krieg, MD, Checkmate Pharmaceuticals Date: 19 April 2018 Description: RNA and DNA oligonucleotides with charged backbones commonly cause immune activation, which can be recognized by appropriate in vitro and/or in vivo testing. Eukaryotic innate immune systems have evolved multiple receptors to ...
Interview with Annabelle Biscans, PhD
Annabelle Biscans, PhD AstraZeneca R&D Sweden How did you become interested in the field of oligonucleotides? My introduction and interest in the oligonucleotide therapeutics world began in 2012 during my PhD at the University of Montpellier, France. When I started my PhD, I did ...
January 2021 Paper of the Month
Koblan LW, Erdos MR, Wilson C, Cabral WA, Levy JM, Xiong ZM, Tavarez UL, Davison LM, Gete YG, Mao X, Newby GA, Doherty SP, Narisu N, Sheng Q, Krilow C, Lin CY, Gordon LB, Cao K, Collins FS, Brown JD, Liu DR. Nature. 2021 Jan;589(7843):608-614.
mRNA Is Capable of Providing Solutions Far Beyond Vaccines
When the FDA issued emergency use authorization for Pfizer-BioNTech and Moderna’s COVID-19 vaccines, mRNA instantly became a household word. Many people believed that this new technology had sprung up out of nowhere in less than a year. What they did not know is that ...
Oligonucleotides Provide Hope for the Treatment of Duchenne Muscular Dystrophy
Imagine if your muscles began weakening for no apparent reason. Then, after an ongoing search for a diagnosis, you learn that you have a disease in which your muscles would progressively waste away and become weaker until you would no longer be able to ...
Can siRNA Therapeutics Heal the Brain?
As we’ve reviewed in past articles the discovery and history of RNA interference and siRNA as a potent therapeutic modality, it brings us to a fascinating idea. Does siRNA carry the potential to heal the brain? If so, can it treat more than genetic ...
