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FDA Draft Guidance – An Exciting Step in the Journey to Bring Oligonucleotide Therapeutics to Patients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as an emerging and promising treatment for rare diseases, the US Food and Drug Administration (FDA) has released a draft guidance for ...
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 a few innovative siRNA medications have been approved to treat ...
Positive Results from Tofersen VALOR Trial and OLE Integrated Data
People diagnosed with the progressive neurodegenerative disease ALS previously had no hope of an effective treatment that could delay the onset of symptoms or extend their lifespan. However, as a result of recent advances in science and medicine, multiple oligonucleotide therapeutics are being developed to provide treatments, one of which is tofersen. Tofersen is an antisense oligonucleotide (ASO) designed ...
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 ...
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 ...
Positive Results from Tofersen VALOR Trial and OLE Integrated Data
People diagnosed with the progressive neurodegenerative disease ALS previously had no hope of an effective ...
