OTS

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 ...

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 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 ...

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 ...

Tofersen, an antisense oligonucleotide treatment for SOD1-ALS, a rare form of amyotrophic lateral sclerosis (ALS), was recently granted accelerated approval by the U.S. FDA. This is quite significant, as approval was granted based on a biomarker rather than clear clinical improvement of the disease. ...