Oligonucleotide Therapeutics Society

Ligand Conjugation – Expanding the Reach of Oligonucleotide Therapeutics

August 25th, 2021|Categories: Perspectives on Current Science|

Oligonucleotide therapies possess enormous potential as healing, life-changing, and cancer-curing drugs. However, there are many barriers to utilizing oligonucleotides to treat disease. Discovering or creating effective delivery methods for each target cell or tissue type solves many of these problems and there are multiple ...

OTS President’s Paper Pick – August 2021

August 9th, 2021|Categories: Perspectives on Current Science, Presidents Pick|

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

August 6th, 2021|Categories: Perspectives on Current Science|

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

August 1st, 2021|Categories: Perspectives on Current Science|

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 ...

Oligonucleotides Provide Hope for the Treatment of Duchenne Muscular Dystrophy

May 20th, 2021|Categories: Perspectives on Current Science|

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 ...

Phase III Trial of ASO Therapy for the Treatment of Huntington’s Disease Halted

April 14th, 2021|Categories: Perspectives on Current Science|

Recently, people who have been impacted by Huntington’s disease, their families, and the OTS community were disappointed to learn that the GENERATION HD1 Phase III study of Tominersen was halted based on the recommendation of an unblinded Independent Data Monitoring Committee. Although not based ...

Go to Top