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.

Bayer to further develop ISIS-FXIRx antisense investigational drug for thrombosis prevention

Bayer HealthCare has entered into an exclusive license agreement with Isis Pharmaceuticals, Inc. on ISIS-FXIRx, an antisense investigational drug in clinical development for the prevention of thrombosis. Under the agreement Bayer will further develop and commercialize ISIS-FXIRx in areas of high unmet medical need.

5 May 2015

Study explores innovative approach to identifying successful treatment for HER2+ breast cancer

Ahmad M. Khalil, PhD, knew the odds were against him -- as in thousands upon thousands to one. Yet he and his team never wavered from their quest to identify the parts of the body responsible for revving up one of the most aggressive forms of breast cancer, HER2+. This month in Breast Cancer Research and Treatment, Khalil and his colleagues at Case Western Reserve University proved the power of persistence; from a pool of more than 30,000 possibilities, they found 38 genes and molecules that most likely trigger HER2+ cancer cells to spread.

25 Apr 2015

Researchers provide new clues, mechanisms to understand functions of 'rebel' DNA in cancer

Genes usually always be expressed as in Western writing: from left to right on the white canvas of our DNA. So when we speak of the activity of our genome, in fact we are referring to the expression of genes in this sense of the double-stranded DNA.

22 Apr 2015

Isis Pharmaceuticals announces positive results from ISIS-ANGPTL3Rx Phase 1 study

Isis Pharmaceuticals, Inc. announced today positive results from a Phase 1 study with ISIS-ANGPTL3Rx. In this study, healthy volunteers treated with ISIS-ANGPTL3Rx achieved dose-dependent, statistically significant reductions in angiopoietin-like 3 (ANGPTL3) of up to 93 percent with a mean reduction of up to 84 percent from baseline (p<0.001).

23 Mar 2015

Researchers reveal how malaria parasite deploys genetic trickery to escape immune system attack

Up to one million people -- mainly pregnant woman and young children -- are killed each year by the Plasmodium falciparum parasite, which causes the most devastating form of human malaria.

26 Feb 2015

Beckman Coulter Genomics Introduces a Scalable RNA-seq service line for processing intact and degraded RNA samples

This fully automated sample preparation pipeline delivers consistent results run after run, reducing user variability and bias and allows processing of a large amount of samples with reduced turnaround time relative to manual library construction.

25 Feb 2015

Isis Pharmaceuticals announces positive results from Phase 1 study of ISIS-PKK Rx for HAE treatment

Isis Pharmaceuticals, Inc. announced today positive results from a Phase 1 study with ISIS-PKKRx. In this study, healthy volunteers treated with ISIS-PKKRx achieved dose-dependent reductions of up to 95 percent in prekallikrein, or PKK. ISIS-PKKRx is a RNA-targeted antisense drug designed to inhibit the production of PKK for the prophylactic treatment of hereditary angioedema (HAE).

24 Feb 2015

Experimental Ebola virus medication shows promise in monkeys

An experimental medication that targets a protein in Ebola virus called VP24 protected 75% of a group of monkeys that were studied from Ebola virus infection, according to new research conducted by the U.S. Army, in collaboration with Sarepta Therapeutics, Inc.

10 Feb 2015

Isis receives $5 million from Biogen Idec to develop drugs for neurological disorders

Isis Pharmaceuticals, Inc. announced today it has earned a $5 million milestone payment from Biogen Idec associated with the validation of an undisclosed target to treat a neurological disorder under its broad strategic collaboration with Biogen Idec to develop drugs to treat patients with neurological disorders.

10 Feb 2015

Vanderbilt researchers find link between the biological clock and Angelman syndrome

Monitoring participants' biological clocks may be the quickest way to determine the effectiveness of experimental drugs currently under development to treat Angelman syndrome: a debilitating genetic disorder that occurs in more than one in every 15,000 live births.

6 Feb 2015

Isis receives $10 million from Biogen Idec to begin IND-supporting studies of ISIS-BIIB4 Rx

Isis Pharmaceuticals, Inc. announced today that it has earned a $10 million milestone payment from Biogen Idec for initiating the investigational new drug (IND)-supporting studies of ISIS-BIIB4 Rx.

5 Feb 2015

Study demonstrates therapeutic potential of new class of synthetic oligonucleotides for DMD treatment

Research efforts associating scientists from the CNRS, UVSQ and INSERM within the Laboratoire END-ICAP, working in collaboration with a team from the University of Bern, has demonstrated the therapeutic potential of a new class of synthetic oligonucleotides in the treatment of Duchenne muscular dystrophy (DMD) using RNA "surgery".

4 Feb 2015

Bern researchers develop active substance for treating Duchenne muscular dystrophy

Duchenne muscular dystrophy is a congenital disease which causes muscle degeneration and eventual death in teenagers. Recently, researchers from Bern developed an active substance, which they together with an international team tested successfully.

3 Feb 2015

Scientists create largest ever catalogue of biological chimeras

Scientists from the Spanish National Cancer Research Centre's Structural Computational Biology Group, led by Alfonso Valencia, are making the largest ever catalogue of biological chimeras available to the public domain.

2 Feb 2015

Researchers gain new insight into how motor neurons in the brain die during ALS

Researchers look to understand the causes of amyotrophic lateral sclerosis (ALS), in the hope of finding new ways to treat the disease. A new study published online today (December 17th) in the Cell Press journal Neuron shows that a common gene mutation in ALS generates a deadly protein that may cause the damage in the brain that leads to ALS.

17 Dec 2014

Isis Pharmaceuticals begins ISIS-DMPK Rx clinical study in DM1 patients

Isis Pharmaceuticals, Inc. announced today that it has initiated a study for ISIS-DMPK Rx in patients with Myotonic Dystrophy Type 1 (DM1). DM1 is a rare genetic neuromuscular disease caused by the production of toxic dystrophia myotonica-protein kinase (DMPK) RNA in cells. ISIS-DMPKRx is specifically designed to reduce toxic DMPK RNA.

16 Dec 2014

ALS Association announces research funds to further understand genetic cause of ALS

The ALS Association is pleased to announce the award of $326,662 in research funds to expand ongoing natural history studies in order to further understand the most common genetic cause of ALS, in preparation for clinical trials in those whose disease is affected by this gene.

14 Dec 2014

Studies presented at ASH meeting compare new, standard-of-care treatments for blood clots

Studies presented at the 56th American Society of Hematology Annual Meeting and Exposition compare new and standard-of-care treatments for blood clots and further illuminate clot risks in vulnerable populations, such as cancer patients.

8 Dec 2014

Isis Pharmaceuticals CEO honored with SCRIP Lifetime Achievement Award

Stanley T. Crooke, Ph.D., M.D., founder, CEO and chairman of the board of directors of Isis Pharmaceuticals, Inc., has been awarded the 2014 SCRIP Lifetime Achievement Award, one of the most prestigious of its kind in the pharmaceutical and biotechnology industries worldwide.

5 Dec 2014

Researchers explore how to turn on the activity of paternal gene

Most genes are inherited as two working copies, one from the mother and one from the father. However, in a few instances, a gene is imprinted, which means that one copy is silenced. This is called genomic imprinting. If the active copy is mutated, then disease results, even though the silenced gene copy may be normal.

2 Dec 2014