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

September 23rd, 2022|Categories: Featured 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: Featured 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 ...

Could Artificial Intelligence Provide the Key to Life Saving Medicines?

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

Drug discovery is time consuming and expensive, requiring trial and error screening. This means that many people have to wait decades, or a lifetime, to find a treatment for their disease. Others, especially those with rare diseases, may never have hope of a treatment. ...

A New ASO Shows Promise in Treating Dravet Syndrome, a Severe Form of Epilepsy

February 28th, 2022|Categories: Featured Perspectives On Current Science, Perspectives on Current Science|

Typically, a single copy of a gene is enough to support normal growth and development but in a small subset of genes, the loss of one copy can cause serious diseases that are often difficult to treat. One of these conditions, Dravet Syndrome, has ...

Clinical Trials Begin for Ultra-Rare Treatment of Alexander Disease

January 1st, 2022|Categories: Featured Perspectives On Current Science, Perspectives on Current Science|

It is always exciting to observe phenomenal results in the treatment of a disease in animal studies, and even more so to see them move into clinical trials. ASOs have proven to be efficient at providing deep reductions in disease causing proteins, and a ...

Ligand Conjugation – Expanding the Reach of Oligonucleotide Therapeutics

August 25th, 2021|Categories: Featured Perspectives On Current Science|

Oligonucleotide therapies possess enormous potential as healing, life-changing, and cancer-curing drugs. However, there are many barriers to utilizing oligonucleotides to treat disease. Discovering or creating effective delivery methods for each target cell or tissue type solves many of these problems and there are multiple ...

A Momentous CRISPR Milestone was Achieved with Remarkable Results

August 1st, 2021|Categories: Perspectives on Current Science, Featured Perspectives On Current Science|

Intellia Therapeutics and Regeneron Pharmaceuticals’ recent announcement is a major milestone worth celebrating. They shared interim data from the first ever human study using systemically administered CRISPR as a gene editing therapy. Early results that surpassed expectations show that not only is the CRISPR-based therapy ...

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