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Targeted antisense oligonucleotide drug tested in humans

January 28, 2019

New Rochelle, NY, January 28, 2019--A first-in-human study with a new class of antisense oligonucleotide therapeutics showed the ability to target the RNA-silencing drug to the liver, resulting in improved potency and safety at therapeutic doses. The design and results of this trial, conducted in healthy human volunteers are reported in Nucleic Acid Therapeutics, a peer-reviewed journal from Mary Ann Liebert, Inc. publishers. Click here to read the full-text article free on the Nucleic Acid Therapeutics website through February 28, 2019.

Stanley Crooke and a team of researchers from Ionis Pharmaceuticals, Carlsbad, CA coauthored the article entitled "Integrated Assessment of the Clinical Performance of GalNAc3-Conjugated 2'-O-Methoxyethyl Chimeric Antisense Oligonucleotides: 1. Human Volunteer Experience." They assessed the safety profile of an antisense oligonucleotide to which had been added a new type of chemical conjugate, N-acetylgalactosamine, or GalNAc3, which selectively targets the systemically administered drug for uptake by the liver. They reported that the conjugated drug was up to 30-fold more potent than the parent antisense oligonucleotide that lacked GalNAc3.

"We applaud and encourage the continued willingness to share such valuable human safety data," says Executive Editor Graham C. Parker, PhD, The Carman and Ann Adams Department of Pediatrics, Wayne State University School of Medicine, Children's Hospital of Michigan, Detroit, MI.
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About the Journal

Nucleic Acid Therapeutics is an authoritative peer-reviewed journal published bimonthly in print and online that focuses on cutting-edge basic research, therapeutic applications, and drug development using nucleic acids or related compounds to alter gene expression. The Journal is under the editorial leadership of Co-Editors-in-Chief Bruce A. Sullenger, PhD, Duke Translational Research Institute, Duke University Medical Center and Annemieke Aartsma-Rus, PhD, Leiden University Medical Center, and Executive Editor Graham C. Parker, PhD. Nucleic Acid Therapeutics is the official journal of the Oligonucleotide Therapeutics Society. Complete tables of content and a sample issue may be viewed on the Nucleic Acid Therapeutics website.

About the Society

The Oligonucleotide Therapeutics Society is an open, non-profit forum to foster academia- and industry-based research and development of oligonucleotide therapeutics. The society brings together the expertise from different angles of oligonucleotide research to create synergies and to bring the field of oligonucleotides to its full therapeutic potential.

About the Publisher

Mary Ann Liebert, Inc., publishers is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research, including Human Gene Therapy, ASSAY and Drug Development Technologies, Applied In Vitro Toxicology, and DNA and Cell Biology. Its biotechnology trade magazine, GEN (Genetic Engineering & Biotechnology News), was the first in its field and is today the industry's most widely read publication worldwide. A complete list of the firm's 80 journals, books, and newsmagazines is available on the Mary Ann Liebert, Inc., publishers website.

Mary Ann Liebert, Inc./Genetic Engineering News

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