Society News

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. This flexible approach to granting approval may have far-reaching implications for the treatment of many ...

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 editing technology created by Harvard and Broad Institute biochemist David Liu? Gene editing has become ...

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 the power to transform the world to the extent that CRISPR and other gene editing ...

CRISPR is now a common term, one that you see even in mainstream media. Yet it is incredible to think that its potential for editing genes was published just ten years ago. In a mere decade, the groundbreaking discovery has been successfully developed to the point that many clinical trials are underway. Although the repeating sequences that make up ...

The chief scientific officer of Ionis Pharmaceuticals, Dr. C. Frank Bennett, is not someone who comes to work just to do science, but to help his patients. Although he started with little knowledge of antisense drugs when he joined the company more than thirty years ago, his patients have remained his greatest source of inspiration in creating life-changing therapies. ...

It’s the middle of winter and, at some point during this season, many people will experience a constellation of symptoms that may include coughing, a sore throat, runny nose, headache, body aches, fevers, and fatigue. Most adults would assume they have a cold or the flu, but now, depending on the exact symptoms experienced, this could also indicate that ...