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 ...
Interview with Steven F. Dowdy, PhD
Steven F. Dowdy, Ph.D.ProfessorDepartment of Cellular & Molecular Medicine,University of California San Diego (UCSD), School of Medicine How did you become interested in the field of oligonucleotides? I started my lab in 1994 as an Investigator of the Howard Hughes Medical Institute (HHMI) and an ...
Can There Be International Agreement on How to Navigate the Future of Genome Editing?
Our world is changing at an astonishingly rapid pace and, as described a few weeks ago, CRISPR is poised to become a major source of change in many fields, from medicine to animals to crops. With any new technology, but especially one that has ...
President’s Pick – March 27 2023
PMID:Â 36881759 DOI:Â 10.1093/nar/gkad067 Writing a good review article is not easy. A good Review not only summarizes the literature, it also provides an expert perspective so that the reader has enough insight to sort the important from the unimportant. That means that the expert authors, who ...









