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 ...

Looking back… Looking forward

December 24th, 2021|Categories: Blog|

This will be my last contribution that I write as President of our Society. On January 1st, 2022, I will become ‘Immediate Past-President’. David Corey (University of Texas, Southwestern) will take over as President and Richard Geary (Ionis Pharmaceuticals) will become the Society’s next ...

IKARIA – An Innovative Platform that Yields Long-Acting siRNA

November 10th, 2021|Categories: Perspectives on Current Science|

In this year that marks the 20th Anniversary of the discovery of RNA interference as a gene-silencing mechanism in mammalian cells, Alnylam announced another remarkable development in siRNA therapeutics – a platform that could allow for administration of siRNA medications just once a year ...

Oligonucleotide Therapies and COVID-19

October 31st, 2021|Categories: Archive|

In response to the COVID-19 pandemic, scientists around the world have joined together in applying their skills and technologies to fight this global threat. Some of the most promising technologies to fight the coronavirus are based on oligonucleotide therapeutics.  In an effort to promote public ...

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