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” oligonucleotides (ASO) designed to bind specifically to complementary “sense” mRNA ...
2022 Annual Meeting Highlights
We truly enjoyed meeting in person again this year and hope those of you who were able to attend enjoyed it as well. We want to share interesting highlights from a few of the sessions. If you missed a talk or want to go back and review any of the speakers, sessions, or posters, they all will remain online ...
Eplontersen May Soon Provide Another Safe, Effective Treatment for People Diagnosed with ATTR
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, fatal disease in which amyloids form when abnormal transthyretin proteins are produced and misfold. The ...
The Personal Journey of Stanley Crooke, the Creation and Development of Ionis, and Beyond
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 perspective of his journey in creating Ionis. But to understand what drove Crooke to ...
A Landmark Permanent Treatment for Children Born with a Deadly Immune Disorder
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 time stem cell gene therapy has treated this specific form of the disease. Severe ...
Treating Cardiovascular Disease by Changing a Single Letter of DNA
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 molecule that causes arteries to accumulate plaque and harden over time. The single-course treatment, called ...
Reflections following the 20th annual meeting on oligonucleotide therapeutics
Footnote from title: modified slightly from “A brief history of oligonucleotide therapeutics” in the Timmerman ...
2022 Annual Meeting Highlights
We truly enjoyed meeting in person again this year and hope those of you who ...
Eplontersen May Soon Provide Another Safe, Effective Treatment for People Diagnosed with ATTR
People who have been diagnosed with hereditary transthyretin amyloidosis (ATTRv) may soon have the option ...
The Personal Journey of Stanley Crooke, the Creation and Development of Ionis, and Beyond
More than 30 years ago, Dr. Stanley Crooke founded Ionis Pharmaceuticals — a biotech company ...
A Landmark Permanent Treatment for Children Born with a Deadly Immune Disorder
In June 2022, the first stem cell gene therapy of Dutch origin was successfully given ...
Treating Cardiovascular Disease by Changing a Single Letter of DNA
A simple spelling change in liver DNA could improve the future of cardiovascular disease treatment. ...
