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” ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
A New Conjugated siRNA provides broad delivery in the CNS, Lung, and Eye
Ever since the groundbreaking discovery of RNA interference and its role as a gene-silencing mechanism in mammalian cells, an ongoing process of discovery has been underway to harness its potential in treating disease. A key mechanism that induces gene-silencing in the RNA pathway is small interfering RNA (siRNA). Although ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
A New Conjugated siRNA provides broad delivery in the CNS, Lung, and Eye
Ever since the groundbreaking discovery of RNA interference and its role as a gene-silencing mechanism in mammalian cells, an ongoing process of discovery has been underway to harness its ...
