Using CRISPR to Personalize Cancer Immunotherapy Treatment

December 12th, 2022|Categories: Featured Perspectives On Current Science, Perspectives on Current Science|

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 ...

Will N-of-1 Drugs Play a Role in the Future of Medicine?

November 22nd, 2022|Categories: Featured Perspectives On Current Science, Perspectives on Current Science|

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 ...

Centyrins Effectively and Safely Deliver siRNAs to Multiple Muscle Tissues

November 9th, 2022|Categories: Featured Perspectives On Current Science, Perspectives on Current Science|

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 ...

Reflections following the 20th annual meeting on oligonucleotide therapeutics

November 3rd, 2022|Categories: Featured Perspectives On Current Science, Perspectives on Current Science|

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 ...

Eplontersen May Soon Provide Another Safe, Effective Treatment for People Diagnosed with ATTR

September 23rd, 2022|Categories: Featured Perspectives On Current Science, Perspectives on Current Science|

People who have been diagnosed with hereditary transthyretin amyloidosis (ATTRv) may soon have the option of choosing another safe, effective therapy, one that significantly improves quality of life, according to patients who received it in the clinical trial. ATTRv is a progressive, debilitating, ...

The Personal Journey of Stanley Crooke, the Creation and Development of Ionis, and Beyond

September 8th, 2022|Categories: Perspectives on Current Science|

More than 30 years ago, Dr. Stanley Crooke founded Ionis Pharmaceuticals — a biotech company that helped pioneer the now successful technology of antisense oligonucleotides (ASO) despite overwhelming challenges and widespread disbelief. After stepping down last year as the company's CEO, Crooke recently published ...

A Landmark Permanent Treatment for Children Born with a Deadly Immune Disorder

August 27th, 2022|Categories: Featured Perspectives On Current Science, Perspectives on Current Science|

In June 2022, the first stem cell gene therapy of Dutch origin was successfully given to a patient by researchers at the Leiden University Medical Centre (LUMC). A baby with severe congenital immune disorder (SCID) received the complex but one-time treatment, marking the first ...

Treating Cardiovascular Disease by Changing a Single Letter of DNA

August 16th, 2022|Categories: Perspectives on Current Science|

A simple spelling change in liver DNA could improve the future of cardiovascular disease treatment. On July 12, biotechnology company Verve Therapeutics announced that the first patient had received a novel gene editing medicine designed to permanently reduce "bad" LDL cholesterol — the fatty ...

FDA Draft Guidance – An Exciting Step in the Journey to Bring Oligonucleotide Therapeutics to Patients

July 26th, 2022|Categories: Perspectives on Current Science|

In recent years, antisense and small interfering RNA (siRNA) oligonucleotide therapeutics have been FDA-approved to treat rare diseases, and many oligonucleotide therapeutics aimed at treating common chronic diseases are in the pipeline. As oligonucleotide therapeutics continue to develop as an emerging and promising treatment for ...

Go to Top