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 remained ...
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 ...
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 ...
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 ...
Will N-of-1 Drugs Play a Role in the Future of Medicine?
Throughout the millennia, anyone who had anything beyond a simple illness could only do their best to ease the pain and petition whatever deities their culture believed in. Then, in the past few centuries, medicine and science have advanced to the point that many illnesses and diseases can be ...
Centyrins Effectively and Safely Deliver siRNAs to Multiple Muscle Tissues
In 2018, Susan Dillon and Karyn O'Neil knew they had created something special that could change the treatment of genetic diseases. Now, their company’s drug that uses their proprietary technology has been granted orphan drug status by the U.S Food and Drug Administration (FDA). The drug, ABX1100, is designed to ...
Reflections following the 20th annual meeting on oligonucleotide therapeutics
Footnote from title: modified slightly from “A brief history of oligonucleotide therapeutics” in the Timmerman Report, copyright 2022, and reprinted with permission. A Brief History of Oligonucleotide Therapeutics About 50 years ago, several scientists independently conceived of the idea of blocking gene expression with synthetic DNA or RNA “antisense” ...
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 started ...
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 ...
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 ...
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% ...
Will N-of-1 Drugs Play a Role in the Future of Medicine?
Throughout the millennia, anyone who had anything beyond a simple illness could only do their best to ease the pain and petition whatever deities their culture believed in. Then, ...
Centyrins Effectively and Safely Deliver siRNAs to Multiple Muscle Tissues
In 2018, Susan Dillon and Karyn O'Neil knew they had created something special that could change the treatment of genetic diseases. Now, their company’s drug that uses their proprietary technology ...
Reflections following the 20th annual meeting on oligonucleotide therapeutics
Footnote from title: modified slightly from “A brief history of oligonucleotide therapeutics” in the Timmerman Report, copyright 2022, and reprinted with permission. A Brief History of Oligonucleotide Therapeutics About ...