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. This flexible approach to granting ...
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 editing technology created by Harvard ...
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 the power to transform the ...
10 Years of CRISPR – Developing a Previously Unimaginable Idea to Benefit the World
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 ...
The Journey of C. Frank Bennett: Caring and Curiosity Drive the Development of Antisense Therapies
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 ...
RSV: Developing Vaccines to Protect the Vulnerable
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 ...
Inclisiran’s Interesting Path to Treating LDL Cholesterol
When The Medicines Company licensed inclisiran, they envisioned a drug for the masses that would save the healthcare system money and be reasonably priced for patients. Despite the company having little experience in the oligonucleotide therapeutics field, it went all in on the drug, selling off its other products and ...
Alnylam’s 20-Year Journey of Discovery
Twenty years ago, a group of scientists intent on developing a new class of medicine using RNAi technology came together and formed Alnylam Pharmaceuticals, a name symbolizing their passion for discovery. The bright center star of Orion's belt, Alnilam, has long been used as a celestial navigator, guiding explorers in ...
New CRISPR Technique May Be the Solution to Replacing Entire Genes
Ever since it was discovered that CRISPR could be used for genome editing, scientists across the world have been working on creating safe, effective ways to use this incredible tool to cure many different human diseases. From the first CRISPR-Cas system that works as genetic scissors to base editing ...
Using CRISPR to Personalize Cancer Immunotherapy Treatment
In 2020, there were an estimated 18.1 million cases of cancer worldwide (excluding non-melanoma skin cancer). Breast, lung, and colorectal cancer were the most common and together contributed to 35.4% of these cases. In the same year, 9.5 million cancer-related deaths were reported. According to the NIH, by 2040, it ...
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 ...
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 ...
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, ...
10 Years of CRISPR – Developing a Previously Unimaginable Idea to Benefit the World
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 ...
The Journey of C. Frank Bennett: Caring and Curiosity Drive the Development of Antisense Therapies
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 ...
RSV: Developing Vaccines to Protect the Vulnerable
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, ...
Inclisiran’s Interesting Path to Treating LDL Cholesterol
When The Medicines Company licensed inclisiran, they envisioned a drug for the masses that would save the healthcare system money and be reasonably priced for patients. Despite the company having ...
Alnylam’s 20-Year Journey of Discovery
Twenty years ago, a group of scientists intent on developing a new class of medicine using RNAi technology came together and formed Alnylam Pharmaceuticals, a name symbolizing their passion for ...
New CRISPR Technique May Be the Solution to Replacing Entire Genes
Ever since it was discovered that CRISPR could be used for genome editing, scientists across the world have been working on creating safe, effective ways to use this incredible ...
Using CRISPR to Personalize Cancer Immunotherapy Treatment
In 2020, there were an estimated 18.1 million cases of cancer worldwide (excluding non-melanoma skin cancer). Breast, lung, and colorectal cancer were the most common and together contributed to 35.4% ...