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 who attended in person or ...
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 that DNA-modifying proteins exist across ...
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 a tumultuous period where Big ...
From Failure to Meet the Clinical Endpoint to U.S. Food and Drug Administration Approval: 15th Antisense Oligonucleotide Therapy Approved Qalsody (Tofersen) for Treatment of SOD1 Mutated Amyotrophic Lateral Sclerosis
On April 25 2023, the U.S. Food and Drug Administration (FDA) granted accelerated approval to tofersen (trade name Qalsody) for the treatment of Superoxide Dismutase 1 (SOD1) associated amyotrophic lateral sclerosis (ALS) [1]. The clinical trial failed to meet its primary endpoint; however, approval was granted based on a ...
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 challenges to be met to ...
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 approval of life-saving drugs ...
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, 2023, in Barcelona, Spain.OTS is ...
Exa-cel: A Potential Breakthrough with Astounding Results
A new therapy that uses the Nobel Prize-winning CRISPR technology may be the latest breakthrough in gene editing therapy. Exagamglogene autotemcel (exa-cel), developed by Vertex Pharmaceuticals and CRISPR Therapeutics, addresses two debilitating blood disorders: sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). If approved, it will mark the world’s first ...
Delivering Oligonucleotide Drugs to the Lungs in a World of Influenza, Coronaviruses, Asthma, and Chronic Lung Diseases
There are many different respiratory viruses, from influenza and respiratory syncytial virus (RSV) to coronaviruses, parainfluenza viruses, rhinoviruses, and adenoviruses. Some are highly pathogenic and can trigger respiratory failure and death. These viruses also facilitate secondary bacterial infections, often resulting in pneumonia, meningitis, and other acute inflammatory pathologies (1). These ...
Martin Egli and Muthiah Manoharan’s Review of Approved Oligonucleotide Therapeutics
Martin Egli and Muthiah Manoharan recently co-wrote an outstanding review that is a great resource for anyone in our community. The review, titled “Chemistry, structure and function of approved oligonucleotide therapeutics,” is a collection of information about the eighteen nucleic acid therapeutics that have been approved in the last ...
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 ...
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 ...
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 ...
From Failure to Meet the Clinical Endpoint to U.S. Food and Drug Administration Approval: 15th Antisense Oligonucleotide Therapy Approved Qalsody (Tofersen) for Treatment of SOD1 Mutated Amyotrophic Lateral Sclerosis
On April 25 2023, the U.S. Food and Drug Administration (FDA) granted accelerated approval to tofersen (trade name Qalsody) for the treatment of Superoxide Dismutase 1 (SOD1) associated amyotrophic ...
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 ...
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 ...
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 ...
Exa-cel: A Potential Breakthrough with Astounding Results
A new therapy that uses the Nobel Prize-winning CRISPR technology may be the latest breakthrough in gene editing therapy. Exagamglogene autotemcel (exa-cel), developed by Vertex Pharmaceuticals and CRISPR Therapeutics, addresses ...
Delivering Oligonucleotide Drugs to the Lungs in a World of Influenza, Coronaviruses, Asthma, and Chronic Lung Diseases
There are many different respiratory viruses, from influenza and respiratory syncytial virus (RSV) to coronaviruses, parainfluenza viruses, rhinoviruses, and adenoviruses. Some are highly pathogenic and can trigger respiratory failure and ...
Martin Egli and Muthiah Manoharan’s Review of Approved Oligonucleotide Therapeutics
Martin Egli and Muthiah Manoharan recently co-wrote an outstanding review that is a great resource for anyone in our community. The review, titled “Chemistry, structure and function of approved ...
