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 ...
FDA Draft Guidance – An Exciting Step in the Journey to Bring Oligonucleotide Therapeutics to Patients
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 rare diseases, the US Food and Drug Administration (FDA) has released a draft guidance for ...
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. ...
FDA Draft Guidance – An Exciting Step in the Journey to Bring Oligonucleotide Therapeutics to Patients
In recent years, antisense and small interfering RNA (siRNA) oligonucleotide therapeutics have been FDA-approved to treat ...
