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 ...
Interview with Sudhir Agrawal, D.Phil.
Sudhir Agrawal, D.Phil. Founder and President, Arnay Sciences Affiliate Professor in the Department of Medicine, UMass Chan Medical School How did you become interested in the field? It was a call from Paul Zamecnik in mid-1986. At the time, I was doing my ...
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 ...
An ASO Is Granted Accelerated Approval By the FDA
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. ...
Pathways for patient-centered interventional genomic medicine
Time: 8AM PST | 11AM EST | 5PM CET Date: June 22, 2023 Speakers: Timothy Yu, PhD, MD Neurogeneticist Boston Children’s Hospital and Harvard Medical School View Bio
Introduction to Nucleic Acid Immunity, and It’s Implications For Optimizing Cancer Immunotherapy
Time: 8AM PST | 11AM EST | 5PM CET Date: June 8, 2023 Starting from the concept and principles of Nucleic Acid Immunity, I will explain why TLR7 and TLR9 are unique among the nucleic acid immune sensing receptors for their ability to ...
President’s Pick – April 2023
Researchers involved in developing oligonucleotide therapeutics have always been known for picking difficult problems related to difficult diseases. SOD1-ALS is a deadly neurodegenerative disease caused by a mutation in the SOD1 protein. Because the disease is due to the SOD1 mutation, it is possible to ...
Innovation, Versatile Gene Editing Technologies, and Patent Battles
Last June, at a genome editing summit in Lisbon, following a much-anticipated presentation by Tessera Therapeutics about their new gene editing technology, an audience member asked a question that is now the basis of a patent dispute: how was it different from the prime ...
