Nav: Home

What's the next step for exon skipping therapies to treat duchenne muscular dystrophy?

October 16, 2017

New Rochelle, NY, October 16, 2017--A team of leading European clinicians and scientists presents a unique perspective on how to move forward in the development of exon skipping therapies to treat the severe muscle-wasting disease Duchenne Muscular Dystrophy (DMD). Following recent approval by the U.S. regulatory authorities of the first such drug for DMD, the team of authors representing key European regulatory agencies, academic medical centers, industry, and patient groups discusses the main challenges and opportunities for the development and regulatory approval of these therapeutic agents going forward, in an article published in Nucleic Acid Therapeutics, a peer-reviewed journal from Mary Ann Liebert, Inc. publishers The article is available free on the Nucleic Acid Therapeutics website.

The article entitled "Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues," was coauthored by Annemieke Aartsma-Rus, PhD, Co-Editor-in-Chief of Nucleic Acid Therapeutics, Pavel Balabanov, MD, PhD, and colleagues from Leiden University Medical Center (The Netherlands), Newcastle University (Newcastle upon Tyne, U.K.), Medicines and Healthcare Product Regulatory Agency (London, U.K.), European Medicines Agency, Bundesinstitut für Arzneimittle und Medizinprodukte (Bonn, Germany), Medicines Evaluation Board (Utrecht, The Netherlands), Catholic University and Centro Clinico Nemo (Rome, Italy), UCL Great Ormond Street Institute of Child Health (London, U.K.), Universidade de Lisboa, Faculdade de Farmácia (Portugal), and United Parent Project Muscular Dystrophy (Amsterdam, The Netherlands).

The authors review the mechanism of action, effectiveness, and limitations of the first-in-class antisense oligonucleotide (AON) drug eteplirsen, which targets exon 51 in patients with DMD. Only 13-14% of patients with DMD have a mutation in exon 51 and would benefit from this treatment, with other small patient clusters requiring distinct AON drugs that each target a different exon. Based on the most recent scientific and medical evidence in the field and the views expressed by European regulators, the authors offer their perspective on the most efficient strategy for developing future exon skipping drugs for DMD, which would deliver effective treatments to patients as quickly as possible. The discussion includes the use of biomarkers in AON drug development, approaches for developing AONS for very small groups of patients, regulatory tools and incentives in the EU, and the potential for obtaining "class" or "platform" approval for AON drugs.

"This paper exemplifies the journal's commitment to advancing the field through consensus, or where necessary, healthy debate among all stakeholders. The piece can also be seen as a call for more candid dialogue with regulatory agencies," 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.
-end-
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 (http://www.liebertpub.com/nat) 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.
140 Huguenot St., New Rochelle
NY 10801-5215
Phone: 914-740-2100
(800) M-LIEBERT
Fax: 914-740-2101http://www.liebertpub.com

Mary Ann Liebert, Inc./Genetic Engineering News

Related Nucleic Acid Therapeutics Articles:

A novel method to precisely deliver therapeutics inside the body
A new way to deliver therapeutic proteins inside the body uses an acoustically sensitive carrier to encapsulate the proteins and ultrasound to image and guide the package to the exact location required, according to Penn State researchers.
New findings shed light on selective therapeutics for IDH1-mutated glioma
Findings of a new study led by Prof. XU Guowang from the Dalian Institute of Chemical Physics (DICP) of the Chinese Academy of Sciences and Prof.
Paving the way for new peptide-based therapeutics with novel method of phage display
Chemists at Texas A&M University are taking a p[h]age from bacteria's playbook in order to beat viruses at their own game and develop new drugs to fight cancer and a host of other human diseases in the process.
New method accelerates development of protein therapeutics
Northwestern Engineering researchers have developed a quick, cell-free system to create biosynthetic pathways to build and study sugar structures.
New insights into the structure and function of Cdc34, a target for cancer therapeutics
Medical University of South Carolina researchers report in Nature Communications they have obtained 3D structural snapshots of Cdc34 in action.
'Green Revolution' in RNAi tools and therapeutics
A team from Nanjing University in China reported that the small silencing RNA sequences against HBsAg generated in edible lettuce (Lactuca sativa L.) can specifically bind and inhibit gene expression in p21-HBsAg knock-in transgenic mice and improve liver injury at a relatively low level when compared to synthetic siRNAs.
Best practices of nucleic acid amplification tests for the diagnosis of clostridioides (clostridium)
A new review looks at the challenges of testing for Clostridioides (Clostridium) difficile infection (CDI) and recommendations for newer diagnostic tests.
Texas A&M research team develops bioinks to print therapeutics in 3D
A team of researchers at Texas A&M University has developed an innovative way to print therapeutics in 3D for regenerative medicine.
New guidelines push for better controlled experiments with synthetic nucleic acids
Researchers have proposed new guidelines to overcome current problems facing scientists developing synthetic nucleic acids -- such as antisense oligonucleotides and double-stranded RNAs -- as drugs and research tools.
Yumanity Therapeutics announces publication of paper in Cell Reports
Yumanity Therapeutics, a company focused on discovering transformative therapies to treat neurodegenerative diseases, today announced the publication of study results describing a potential new target for therapeutic intervention in Parkinson's disease and other related disorders.
More Nucleic Acid Therapeutics News and Nucleic Acid Therapeutics Current Events

Trending Science News

Current Coronavirus (COVID-19) News

Top Science Podcasts

We have hand picked the top science podcasts of 2020.
Now Playing: TED Radio Hour

Listen Again: Meditations on Loneliness
Original broadcast date: April 24, 2020. We're a social species now living in isolation. But loneliness was a problem well before this era of social distancing. This hour, TED speakers explore how we can live and make peace with loneliness. Guests on the show include author and illustrator Jonny Sun, psychologist Susan Pinker, architect Grace Kim, and writer Suleika Jaouad.
Now Playing: Science for the People

#565 The Great Wide Indoors
We're all spending a bit more time indoors this summer than we probably figured. But did you ever stop to think about why the places we live and work as designed the way they are? And how they could be designed better? We're talking with Emily Anthes about her new book "The Great Indoors: The Surprising Science of how Buildings Shape our Behavior, Health and Happiness".
Now Playing: Radiolab

The Third. A TED Talk.
Jad gives a TED talk about his life as a journalist and how Radiolab has evolved over the years. Here's how TED described it:How do you end a story? Host of Radiolab Jad Abumrad tells how his search for an answer led him home to the mountains of Tennessee, where he met an unexpected teacher: Dolly Parton.Jad Nicholas Abumrad is a Lebanese-American radio host, composer and producer. He is the founder of the syndicated public radio program Radiolab, which is broadcast on over 600 radio stations nationwide and is downloaded more than 120 million times a year as a podcast. He also created More Perfect, a podcast that tells the stories behind the Supreme Court's most famous decisions. And most recently, Dolly Parton's America, a nine-episode podcast exploring the life and times of the iconic country music star. Abumrad has received three Peabody Awards and was named a MacArthur Fellow in 2011.