RNA Single-base Editing Therapy that Treats Genetic Lung and Liver Disease Entered Clinical Trials
When Peggy's mom was in her forties, she started to have trouble breathing. Although she had never smoked, she had been exposed second-hand earlier in her life. Eventually, her mother ended up at the Mayo Clinic, where she was the 36th patient to be ...
Jennifer Doudna – Seeking to Improve the World with CRISPR
At 10:53 p.m. on Oct. 7, Jennifer Doudna woke to a buzzing sound. Picking up her phone — which she noticed had multiple missed calls and messages — she was greeted by the voice of Nature journalist Heidi Ledford. The day before, Doudna had ...
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 ...
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, ...
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 ...
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 ...
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 ...
Amazon Prime for DNA – Has A New Era of Oligonucleotide Synthesis Begun?
Phosphoramidite chemistry has been used for decades to write synthetic DNA, but challenges arise when longer, more complex oligos are needed. After years of innovation fueled by the goal of meeting this need, Ansa Biotechnologies announced de novo synthesis of a 1005 base long oligonucleotide ...
