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 [...]
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. [...]
President’s Pick of Presentations from the Annual OTS Meeting
By: Annemieke Aartsma-Rus, Ph.D.The second virtual annual meeting of the Oligonucleotide Therapeutics Society (OTS) took place September 26-29 on a computer or laptop near you. Despite the challenges the pandemic poses on research and development in general, the oligonucleotide therapy field has made many advances, [...]
2021 Annual Meeting Highlights
We enjoyed another excellent Annual Meeting this year! A more extensive meeting review from the organizing committee will be forthcoming, so be sure to watch for it. In the meantime, here is a broad overview highlighting a few of the many fascinating and intriguing [...]
Non-Profits Create Next-Generation Medicines that Treat Ultra-Rare Diseases
Hundreds of millions of people worldwide are living with a rare disease. Most rare diseases do not currently have a cure. So, once diagnosed with a rare disease, most patients and their families are merely given strategies to manage the disease symptoms and try [...]
Oligonucleotides Safely Cross the Blood-Brain Barrier to Knock Down Gene Expression
Neurodegenerative diseases, brain tumors, and infectious diseases and inflammatory conditions that impact the brain wreak havoc on a person’s health and are notoriously difficult to treat. Oligonucleotide drugs could provide solutions to many pathological brain conditions if certain challenges could be overcome. Researchers at Tokyo [...]
OTS President’s Paper Pick – August 2021
By: Annemieke Aartsma-Rus, Ph.D. The paper information: CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis Julian D Gillmore, Ed Gane, Jorg Taube, Justin Kao, Marianna Fontana, Michael L Maitland, Jessica Seitzer, Daniel O’Connell, Kathryn R Walsh, Kristy Wood, Jonathan Phillips, Yuanxin Xu, Adam Amaral, Adam [...]
Lipid Nanoparticles: Nanomedicine’s Triumph
Oligonucleotide therapeutics have been making headlines this past year, between the groundbreaking new mRNA vaccines and the recent news of phenomenal interim results for Intellia’s investigational genome editing treatment for ATTR amyloidosis. Neither of these would have been possible without a lesser-known component – [...]
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 therapy [...]
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 [...]