Oligonucleotide Therapeutics Society

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 [...]

A Potent ASO Shows Encouraging Results in A Single-Patient Pilot Study for ALS

February 9th, 2022|Categories: Perspectives on Current Science|

Amyotrophic lateral sclerosis (ALS) is a progressive, neurodegenerative disease in which motor neurons die. Since the motor neurons that die control voluntary muscles, the disease causes great difficulty for those who must live with it. They will experience loss of motor control of the [...]

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 [...]

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 [...]

New Miniature CRISPR-Cas Systems Engineered to Function in Human Cells

October 28th, 2021|Categories: Perspectives on Current Science|

CRISPR-Cas systems provide a revolutionary approach to curing disease through precise genome editing and CRISPR therapies have delivered promising early successes. However, engineering more highly efficient, compact Cas systems could result in a more versatile tool that could greatly improve and expand CRISPR-based therapeutics. [...]

President’s Pick of Presentations from the Annual OTS Meeting

October 26th, 2021|Categories: Presidents Pick, Perspectives on Current Science|

By: Annemieke Aartsma-Rus, Ph.D. The second virtual annual meeting of the Oligonucleotide Therapeutics Society (OTS) took place September 26-29 on a computer or laptop near you. Despite the challenges the pandemic poses on research and development in general, the oligonucleotide therapy field has made [...]

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