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
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 [...]
2021 Dr. Alan M. Gewirtz Memorial Scholarship Award for Postdoctoral Fellows and Junior Industrial Professionals
Kotaro Yoshioka, MD, PhD Department of Neurology and Neurological Science, Tokyo Medical and Dental University Biography: Kotaro Yoshioka received his PhD in 2016 from Tokyo Medical and Dental University (Japan), where he has developed a unique double-stranded ASO technology, “Heteroduplex oligonucleotide (HDO)” in the laboratory [...]
2021 Dr. Alan M. Gewirtz Memorial Scholarship Award for Graduate Students Winner Mr. Hassan Fakih
Mr. Hassan Fakih Department of Chemistry, McGill University Biography: Hassan obtained his bachelor’s degree in chemistry from the American University of Beirut in 2016. Then, he Joined the Sleiman Lab at the Chemistry department of McGill University to pursue his doctorate degree. His work [...]
New Miniature CRISPR-Cas Systems Engineered to Function in Human Cells
CRISPR-Cas systems provide a revolutionary approach to curing disease through precise genome editing and CRISPR therapies have delivered promising early successes. However, engineering more highly efficient, compact Cas systems could result in a more versatile tool that could greatly improve and expand CRISPR-based therapeutics. [...]
