Looking back… Looking forward
This will be my last contribution that I write as President of our Society. On January 1st, 2022, I will become ‘Immediate Past-President’. David Corey (University of Texas, Southwestern) will take over as President and Richard Geary (Ionis Pharmaceuticals) will become the Society’s next ...
Can an ASO Reduce Disease Progression in ALS?
What if you noticed your body acting a bit “off,” doing something unusual and more than a bit worrying? Then, you go to a doctor and are eventually told that you have a debilitating neurodegenerative disease and can expect to progressively lose control of your ...
Interview with Vasant Jadhav, PhD
Vasant Jadhav, PhD. SVP Head of RNAi Platform Alnylam Pharmaceuticals How did you become interested in the field of oligonucleotides and who were your early mentors? During undergrad, I was intrigued by the molecular machines and mechanisms behind secrets of life- replication, transcription, ...
IKARIA – An Innovative Platform that Yields Long-Acting siRNA
In this year that marks the 20th Anniversary of the discovery of RNA interference as a gene-silencing mechanism in mammalian cells, Alnylam announced another remarkable development in siRNA therapeutics – a platform that could allow for administration of siRNA medications just once a year ...
Development of N=1 RNA Therapies in the Netherlands and Europe
After the development of Milasen in the United States, there has been an increase in the development of antisense oligonucleotide therapies for patients with unique mutations.
Oligonucleotide Therapies and COVID-19
In response to the COVID-19 pandemic, scientists around the world have joined together in applying their skills and technologies to fight this global threat. Some of the most promising technologies to fight the coronavirus are based on oligonucleotide therapeutics. In an effort to promote public ...
2021 Late Discovery Paper Of The Year Award – Winners
An mRNA Vaccine Against SARS-CoV-2 – Preliminary Report Phase I/II study of COVID-19 RNA vaccine BNT 162b1 in adults The need for platforms to develop vaccines rapidly and efficiently was clearly highlighted by the Covid-19 pandemic. The first vaccine against SARS-CoV-2 was approved only ...
2021 Basic Paper Of The Year Award – Winners
Investigating the pharmacodynamic durability of GalNAc-siRNA conjugates Givosiran, a GalNAc-conjugated siRNA, which was recently approved on the market, displays sustained knock-down over month-long periods. The following paper investigates the reasons behind those impressive pharmacodynamic properties. After looking into various mechanisms related to stability, accumulation or ...
2021 Mary Ann Liebert Publishers, Inc. Young Investigator Award Winner Alex Garanto, PhD
Alex Garanto, PhD Radboud University Medical Center (Radboudumc) Biography: Dr. Alejandro (Alex) Garanto is currently an Assistant Professor at the Departments of Pediatrics and Human Genetics of the Radboudumc in Nijmegen (the Netherlands). He obtained his PhD from the University of Barcelona in 2011 ...
