Antisense News and Research

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Antisense is the non-coding strand in double-stranded DNA. The antisense strand serves as the template for mRNA synthesis.

Ico Therapeutics completes Phase 2 iDEAL study for treatment of diabetic macular edema

iCo Therapeutics Inc. announced today that it has completed enrollment of its Phase 2 iDEAL study evaluating the efficacy and safety after repeated injections of iCo-007 in patients with Diabetic Macular Edema (DME). The study has shown a good safety profile with no drug-related serious adverse events to date.

19 Jun 2013

Scientists find that 'junk DNA' could promote cancer progression

Scientists at The University of Nottingham have found that a genetic rogue element produced by sequences until recently considered 'junk DNA' could promote cancer progression.

30 May 2013

Isis begins clinical study of ISIS-STAT3 in patients with metastatic liver cancer

Isis Pharmaceuticals, Inc. announced the initiation of a Phase 1b/2a clinical study of ISIS-STAT3Rx in patients with advanced metastatic hepatocellular carcinoma (HCC).

7 May 2013

Novel strategy for characterizing RNAs transported from nerve cell to synapse

Despite decades of research, relatively little is known about the identity of RNA molecules that are transported as part of the molecular process underpinning learning and memory.

26 Apr 2013

Investigational treatment for ALS passes early phase clinical trial for safety

An investigational treatment for an inherited form of Lou Gehrig's disease has passed an early phase clinical trial for safety, researchers at Washington University School of Medicine in St. Louis and Massachusetts General Hospital report.

24 Apr 2013

Isis Pharmaceuticals starts ISIS-SMNRx Phase 2 study in infants with SMA

Isis Pharmaceuticals, Inc. announced the initiation of a Phase 2 study of ISIS-SMNRx in infants with spinal muscular atrophy. SMA is a severe and rare genetic neuromuscular disease characterized by muscle atrophy and weakness and is the most common genetic cause of infant mortality.

24 Apr 2013

Xenon Pharmaceuticalsn receives BIOTECanada's Gold Leaf Company of the Year Award for 2013

Xenon Pharmaceuticals Inc. is pleased to announce that it has been awarded BIOTECanada's Gold Leaf Company of the Year Award for 2013.

22 Apr 2013

Santaris Pharma, Bristol-Myers Squibb partner to develop novel medicines using LNA Drug Platform

Santaris Pharma A/S, a privately held biopharmaceutical company focused on developing medicines targeted to disease-related mRNAs and microRNAs, today announced a worldwide strategic alliance with Bristol-Myers Squibb to discover and develop novel medicines using Santaris Pharma's proprietary Locked Nucleic Acid Drug Platform.

18 Apr 2013

Roche, Isis Pharmaceuticals to develop neurodegenerative therapeutics for Huntington's disease

Roche and Isis Pharmaceuticals, Inc today announced that they have formed an alliance to develop treatments for Huntington's disease based on Isis' antisense oligonucleotide technology.

9 Apr 2013

Clinical trial signals new era in treatment of neurodegererative disorders

The initial clinical trial of a novel approach to treating amyotrophic lateral sclerosis - blocking production of a mutant protein that causes an inherited form of the progressive neurodegererative disease - may be a first step towards a new era in the treatment of such disorders.

4 Apr 2013

Isis Pharmaceuticals starts ISIS-APOARx Phase 1 clinical study for treatment of atherosclerosis

Isis Pharmaceuticals, Inc. announced today that it has initiated a Phase 1 clinical study for ISIS-APOARx, an antisense drug targeting apolipoprotein(a) for the treatment of atherosclerosis.

3 Apr 2013

Two studies shed light on molecular biology of three blood disorders

Two studies led by investigators at Weill Cornell Medical College shed light on the molecular biology of three blood disorders, leading to novel strategies to treat these diseases.

26 Mar 2013

Columbia University Medical Center, NewYork-Presbyterian Hospital present research works at AAN meeting

The following research from Columbia University Medical Center and NewYork-Presbyterian Hospital is being presented at the 65th annual meeting of the American Academy of Neurology, March 16-23, 2013, in San Diego.

18 Mar 2013

Systemic delivery of antisense oligonucleotides neutralizes mutant RNA toxicity in mice with DM1

By targeting the specific mutation that causes the hereditary neuromuscular disease myotonic dystrophy, it is possible to neutralize the mutant RNA toxicity and minimize or even eliminate the disabling symptoms of the disease.

25 Feb 2013

Non-coding RNA MALAT1 influences lung cancer metastasis

The vast majority - approximately 80 percent - of our DNA does not code for proteins, yet it gets transcribed into RNA. These RNA molecules are called non-coding and fulfill multiple tasks in the cell. Alongside a well-studied group of small RNAs, there is also a class of so-called long non-coding RNAs consisting of more than 200 nucleotides.

6 Feb 2013

Two new methods could accelerate development of improved antisense drugs

Powerful antisense drugs that target disease-associated genes to block their expression can be used to treat a broad range of diseases. Though antisense therapy has been proven effective, challenges remain in ensuring that the drugs reach their intended targets.

2 Feb 2013

FDA approves Genzyme’s KYNAMRO to treat homozygous familial hypercholesterolemia

Genzyme, a Sanofi company, and Isis Pharmaceuticals Inc., today announced that the U.S. Food and Drug Administration (FDA) has approved its New Drug Application (NDA) for KYNAMROTM (mipomersen sodium) injection.

30 Jan 2013

Study details exactly how Her2 gene promotes progression and spread of breast cancer cells

Research led by Dr. Suresh Alahari, the Fred Brazda Professor of Biochemistry and Molecular Biology at LSU Health Sciences Center New Orleans and its Stanley S. Scott Cancer Center, details exactly how the Her2 cancer gene promotes the progression and spread of breast cancer cells.

23 Jan 2013

Scientists design small molecules that recognize myotonic dystrophy-associated RNAs

Scientists from the Florida campus of The Scripps Research Institute (TSRI) have developed a new approach to alter the function of RNA in living cells by designing molecules that recognize and disable RNA targets. As a proof of principle, in the new study the team designed a molecule that disabled the RNA causing myotonic dystrophy.

21 Dec 2012

Dantrolene shows promise for treating DMD

A muscle relaxant called dantrolene used for treating malignant hyperthermia boosts the activity of therapies currently being developed to treat the genetic disorder Duchenne muscular dystrophy (DMD), show study findings.

17 Dec 2012