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Isis Pharmaceuticals begins ISIS-DMPK Rx Phase 1 study for treatment of Myotonic Dystrophy Type 1

Isis Pharmaceuticals begins ISIS-DMPK Rx Phase 1 study for treatment of Myotonic Dystrophy Type 1

Isis Pharmaceuticals, Inc. announced today that it has initiated a Phase 1 study for ISIS-DMPKRx. Isis earned a $14 million milestone payment from Biogen Idec associated with this achievement. [More]
Isis Pharmaceuticals reports pro forma net operating loss of $22.6 million for Q1 2014

Isis Pharmaceuticals reports pro forma net operating loss of $22.6 million for Q1 2014

Isis Pharmaceuticals, Inc. today reported a pro forma net operating loss (NOL) of $22.6 million for the three months ended March 31, 2014 compared to pro forma operating income of $4.5 million for the same period in 2013. [More]
Researchers use RNA sequencing to look at early- and late-stage development of the heart

Researchers use RNA sequencing to look at early- and late-stage development of the heart

Most people think the development of the heart only happens in the womb, however the days and weeks following birth are full of cellular changes that play a role in the structure and function of the heart. Using mouse models, researchers at Baylor College of Medicine have now been able to categorize the alternative splicing (the process in which genes code proteins, determining their role) that takes place during these changes and what mechanisms they affect. [More]
U.Va. researchers named recipients of 2013 Hartwell Individual Biomedical Research Awards

U.Va. researchers named recipients of 2013 Hartwell Individual Biomedical Research Awards

University of Virginia neurologist Dr. Erin Pennock Foff, biologist Sarah Kucenas and biomedical engineer Shayn Peirce-Cotter have been named recipients of 2013 Hartwell Individual Biomedical Research Awards to benefit children of the United States. Each scientist will receive $100,000 in direct annual research support from The Hartwell Foundation for three years. [More]
Isis Pharmaceuticals provides update on ISIS-SMNRx Phase 2 study in infants with SMA

Isis Pharmaceuticals provides update on ISIS-SMNRx Phase 2 study in infants with SMA

Isis Pharmaceuticals, Inc. today provided an update on its ongoing open label, multiple dose Phase 2 study of ISIS-SMNRx in infants with spinal muscular atrophy (SMA). [More]
Scientists reveal atomic-level view of genetic defect that causes myotonic dystrophy type 2

Scientists reveal atomic-level view of genetic defect that causes myotonic dystrophy type 2

Scientists from the Florida campus of The Scripps Research Institute have revealed an atomic-level view of a genetic defect that causes a form of muscular dystrophy, myotonic dystrophy type 2, and have used this information to design drug candidates with potential to counter those defects—and reverse the disease. [More]
Marina Biotech, Mirna modify license agreement regarding development of microRNA-based therapeutics

Marina Biotech, Mirna modify license agreement regarding development of microRNA-based therapeutics

Marina Biotech, Inc., a leading oligonucleotide-based drug discovery and development company, and Mirna Therapeutics, Inc., a privately-held biotechnology company pioneering microRNA replacement therapy for cancer, announced today that they have amended their license agreement regarding the development and commercialization of microRNA-based therapeutics utilizing Mirna's proprietary microRNAs and Marina Biotech's novel SMARTICLES liposomal delivery technology. [More]
Stem cell lines are ideal research tools for designing models to understand disease progression

Stem cell lines are ideal research tools for designing models to understand disease progression

Scientists from King's College London have announced that 16 human embryonic stem (hES) cell lines have been approved by the US National Institutes of Health (NIH) and placed on their Stem Cell Registry, making them freely available for federally-funded research in the USA. [More]
Researchers identify likely molecular pathway that causes untreatable neurodegenerative diseases

Researchers identify likely molecular pathway that causes untreatable neurodegenerative diseases

University of Adelaide researchers have identified a likely molecular pathway that causes a group of untreatable neurodegenerative diseases, including Huntington's disease and Lou Gehrig's disease. [More]
Scientists identify RNA regulator that plays critical role in proper functioning of hESCs

Scientists identify RNA regulator that plays critical role in proper functioning of hESCs

Scientists at A*STAR's Genome Institute of Singapore, in collaboration with their counterparts from Canada, Hong Kong and US, have discovered a protein mediator SON plays a critical role in the health and proper functioning of human embryonic stem cells. This finding was reported on 8th September 2013 in the advanced online issue of the prestigious science journal Nature Cell Biology. [More]
Biogen Idec And Isis Ink Fourth Deal In Two Years

Biogen Idec And Isis Ink Fourth Deal In Two Years

... [More]
Novel way to dramatically raise potency of drug candidates targeting RNA

Novel way to dramatically raise potency of drug candidates targeting RNA

Scientists from the Jupiter campus of The Scripps Research Institute (TSRI) have shown a novel way to dramatically raise the potency of drug candidates targeting RNA, resulting in a 2,500-fold improvement in potency and significantly increasing their potential as therapeutic agents. [More]
TSRI scientists identify small molecules to control genetic defect responsible for muscular dystrophy

TSRI scientists identify small molecules to control genetic defect responsible for muscular dystrophy

For the first time, scientists from the Florida campus of The Scripps Research Institute have identified small molecules that allow for complete control over a genetic defect responsible for the most common adult onset form of muscular dystrophy. [More]
New hope for patients with myotonic dystrophy

New hope for patients with myotonic dystrophy

There's hope for patients with myotonic dystrophy. A new small molecule developed by researchers at the University of Illinois has been shown to break up the protein-RNA clusters that cause the disease in living human cells, an important first step toward developing a pharmaceutical treatment for the as-yet untreatable disease. [More]
Clinical trial signals new era in treatment of neurodegererative disorders

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. [More]

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. [More]
Scientists design small molecules that recognize myotonic dystrophy-associated RNAs

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. [More]
Isis, Biogen Idec partner to discover and develop antisense drugs against neuromuscular disorders

Isis, Biogen Idec partner to discover and develop antisense drugs against neuromuscular disorders

Biogen Idec and Isis Pharmaceuticals, Inc. today announced that they have entered into a global collaboration agreement under which the companies will discover and develop antisense drugs against three undisclosed targets to treat neurological or neuromuscular disorders. [More]
Researchers hold new implications for pathogenesis of myotonic dystrophy

Researchers hold new implications for pathogenesis of myotonic dystrophy

An important breakthrough could help in the fight against myotonic dystrophy. The discovery, recently published in the prestigious scientific journal Cell, results from an international collaboration between researchers at the IRCM, the Massachusetts Institute of Technology (MIT), the University of Southern California and Illumina. [More]
Scripps Florida designs new compound that shows promise against tremor ataxia syndrome

Scripps Florida designs new compound that shows promise against tremor ataxia syndrome

Scientists on the Florida campus of The Scripps Research Institute have designed a compound that shows promise as a potential therapy for one of the diseases closely linked to fragile X syndrome, a genetic condition that causes mental retardation, infertility, and memory impairment, and is the only known single-gene cause of autism. [More]