FDA Approved the First CRISPR Treatment – Potential Cure for People with Sickle Cell Disease
Victoria Gray was diagnosed with sickle cell disease (SCD) when she was just three months old; since then, episodes of pain and frequent hospitalizations have been a part of her life. Many of her dreams seemed like far-off impossibilities when the smallest things, like ...
Follow-up Data Confirms That Inclisiran Provides Long-Term Reduction of LDL-Cholesterol
Cardiovascular disease is the leading cause of death in the United States, exceeding all types of cancer, unintentional injury, and stroke combined. However, updated data from Novartis's open-label trial shows promising long-term results for inclisiran (Leqvio), the first and only small interfering RNA (siRNA) ...
The First FDA Approval for a GalNAc-conjugated ASO
It starts with a pins-and-needle sensation in your feet, or maybe your gastrointestinal tract has become easily irritated, or you've started to lose weight or develop heart problems. Hereditary ATTR amyloidosis can present differently among patients and with symptoms found in far more common ...
Base Editing in Clinical Trials to Treat Acute Lymphoblastic Leukemia
Base editing has experienced a rapid rise in use since it first came on the scene in 2016, with multiple trials underway, testing its ability to treat conditions with precise, single-letter changes to DNA. The technique presents a potentially more accurate and safer method ...
2023 Annual Meeting Highlights
We are pleased to note that this year’s annual Oligonucleotide Therapeutics Society meeting in Barcelona, Spain had the highest involvement of any previous meeting, with 800 in-person attendees, 297 abstract submissions, 260 posters, 47 travel grants, 36 Sponsors, and 29 Exhibitors. We hope all ...
Fanzor: A Programmable RNA-Guided System In Eukaryotes Similar to CRISPR
Researchers have uncovered the first programmable RNA-guided system in eukaryotes that could be even more precise than CRISPR gene-editing. The discovery — led by Feng Zhang at the McGovern Institute for Brain Research at MIT and the Broad Institute of MIT and Harvard — demonstrates ...
RNAi: The Persistence of a Few Leads to Success
On August 10, 2018, the U.S. Food and Drug Administration (FDA) approved the first RNAi therapy. The approval marked a significant milestone not just for the drug but the entire field of RNA interference (RNAi). The success of the technology was never guaranteed, and ...
Investigating the Biology and Potential of CircRNA
In the past few years, mRNA has become a mainstream word with the swift creation of mRNA vaccines to treat SARS-CoV-2. This new technology was already in development, placing it in the perfect position to meet an urgent global need. Yet, there are still ...
Hydrogels and Nucleic Acids – An Innovative Combination
An incredible amount of time, effort, and money have gone into finding increasingly better ways to safely and effectively deliver nucleic acid based therapeutics. Many solutions have been developed that do indeed provide safe and effective delivery, and we have had many occasions to celebrate ...
The Oligonucleotide Therapeutics Society is Pleased to Announce the 2023 Annual Meeting in Barcelona, Spain
The Oligonucleotide Therapeutics Society (OTS) is excited to bring together those in the field of oligonucleotide therapeutics for its 19th Annual Oligo Meeting. Anyone interested in oligonucleotide therapeutics is welcome to join the event, which will be held virtually and in person on Oct. 22-25, ...
