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Antisense is the non-coding strand in double-stranded DNA. The antisense strand serves as the template for mRNA synthesis.
New consensus guidelines for toxicity testing of oligonucleotide-based therapeutics

New consensus guidelines for toxicity testing of oligonucleotide-based therapeutics

Oligonucleotide-based therapeutics present unique challenges when it comes to testing their potential to cause reproductive and developmental harm. New consensus guidelines for toxicity testing that take into consideration the combined chemical and biological characteristics of these novel biopharmaceuticals are presented in Nucleic Acid Therapeutics, a peer-reviewed journal from Mary Ann Liebert, Inc. publishers. [More]
Isis Pharmaceuticals provides update on Phase 2 clinical studies of ISIS-SMN Rx in children with SMA

Isis Pharmaceuticals provides update on Phase 2 clinical studies of ISIS-SMN Rx in children with SMA

Isis Pharmaceuticals, Inc. today provided an update on its ongoing open-label Phase 2 clinical studies of ISIS-SMN Rx in infants and children with spinal muscular atrophy at the 19th International World Muscle Society (WMS) Congress in Berlin, Germany. [More]
Isis Pharmaceuticals secures $18 million from GSK to advance ISIS-TTR Rx Phase 2/3 study

Isis Pharmaceuticals secures $18 million from GSK to advance ISIS-TTR Rx Phase 2/3 study

Isis Pharmaceuticals, Inc. announced today that it has earned an $18 million milestone payment from GlaxoSmithKline related to the advancement of the Phase 2/3 study of ISIS-TTRRx in patients with familial amyloid polyneuropathy (FAP). [More]
GSK awards milestone payment to Isis Pharmaceuticals to develop ISIS-GSK4Rx for ocular disease

GSK awards milestone payment to Isis Pharmaceuticals to develop ISIS-GSK4Rx for ocular disease

Isis Pharmaceuticals, Inc. announced today that it has earned a $1.5 million milestone payment from GSK related to the advancement of the program to develop ISIS-GSK4Rx to treat an undisclosed ocular disease. [More]
Isis receives $4 million in milestone payment from Achaogen for initiation of plazomicin Phase 3 study

Isis receives $4 million in milestone payment from Achaogen for initiation of plazomicin Phase 3 study

Isis Pharmaceuticals, Inc. announced that it has earned a $4 million milestone payment from Achaogen, Inc. associated with the initiation of a Phase 3 study of plazomicin in patients with serious multi-drug resistant (MDR), gram-negative bacterial infections. [More]
MU researchers make new breakthrough in spinal muscular atrophy drug

MU researchers make new breakthrough in spinal muscular atrophy drug

According to recent studies, approximately one out of every 40 individuals in the United States is a carrier of the gene responsible for spinal muscular atrophy (SMA), a neurodegenerative disease that causes muscles to weaken over time. [More]
ANP reports primary efficacy results from ATL1103 Phase II trial in patients with acromegaly

ANP reports primary efficacy results from ATL1103 Phase II trial in patients with acromegaly

Antisense Therapeutics Limited is pleased to report the primary efficacy results from its Phase II clinical trial of ATL1103 in patients with the potentially life threatening growth disorder, acromegaly. [More]
Isis Pharmaceuticals begins ISIS-APOCIIIRx Phase 3 study in FCS patients

Isis Pharmaceuticals begins ISIS-APOCIIIRx Phase 3 study in FCS patients

Isis Pharmaceuticals, Inc. announced the initiation of a Phase 3 study evaluating ISIS-APOCIIIRx in patients with familial chylomicronemia syndrome (FCS). FCS is a rare orphan disease, characterized by extremely high triglyceride levels, that affects an estimated 3,000 to 5,000 patients worldwide. [More]
Research shows long non-coding RNAs regulate circadian clocks

Research shows long non-coding RNAs regulate circadian clocks

Researchers at UT Southwestern Medical Center have found a new way that internal body clocks are regulated by a type of molecule known as long non-coding RNA. [More]
Isis earns $1 million payment from GSK related to advancement of ISIS-TTRRx

Isis earns $1 million payment from GSK related to advancement of ISIS-TTRRx

Isis Pharmaceuticals, Inc. announced today that it has earned a $1 million milestone payment from GlaxoSmithKline (GSK) related to the advancement of the Phase 2/3 study of ISIS-TTRRx in patients with familial amyloid polyneuropathy (FAP). [More]
Marginal division of striatum plays similar function in learning and memory to hippocampus

Marginal division of striatum plays similar function in learning and memory to hippocampus

In addition to the hippocampus, the marginal division of the striatum is also involved in learning and memory. [More]
Isis Pharmaceuticals gets $1 million from GSK to discover and develop drugs for HBV

Isis Pharmaceuticals gets $1 million from GSK to discover and develop drugs for HBV

Isis Pharmaceuticals, Inc. announced today that it has earned a $1 million payment from GlaxoSmithKline related to the advancement of its program to develop antisense drugs to treat hepatitis B virus (HBV). [More]
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]
New compound restores learning, memory and other symptoms of AD in mice

New compound restores learning, memory and other symptoms of AD in mice

A molecular compound developed by Saint Louis University scientists restored learning, memory and appropriate behavior in a mouse model of Alzheimer's disease, according to findings in the May issue of the Journal of Alzheimer's Disease. [More]
Isis Pharmaceuticals starts Phase 1 clinical study of ISIS-PKKRx to treat patients with HAE

Isis Pharmaceuticals starts Phase 1 clinical study of ISIS-PKKRx to treat patients with HAE

Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it initiated a Phase 1 clinical study of ISIS-PKKRx. ISIS-PKKRx is an antisense drug in development to treat patients with hereditary angioedemia (HAE). [More]
Bio-Path Holdings reports net loss of $504,876 for first quarter 2014

Bio-Path Holdings reports net loss of $504,876 for first quarter 2014

Bio-Path Holdings, Inc., a biotechnology company developing a liposomal delivery technology for nucleic acid cancer drugs, today announced operational and financial results for the first quarter ended March 31, 2014. [More]

Isis Pharmaceuticals receives $3 million milestone payment from GSK

Isis Pharmaceuticals, Inc. announced today that it has earned a $3.0 million milestone payment from GlaxoSmithKline (GSK) related to the initiation of a natural history study, which will support the advancement of one of the alliance programs. [More]
Isis Pharmaceuticals reports positive data from ISIS-GCGRRx Phase 2 study in patients with type 2 diabetes

Isis Pharmaceuticals reports positive data from ISIS-GCGRRx Phase 2 study in patients with type 2 diabetes

Isis Pharmaceuticals, Inc. announced today positive data from a Phase 2 study of ISIS-GCGRRx in patients with type 2 diabetes uncontrolled on stable metformin therapy. [More]
Genzyme, Isis receive 2014 Partners in Progress Corporate Award for KYNAMRO injection

Genzyme, Isis receive 2014 Partners in Progress Corporate Award for KYNAMRO injection

Genzyme, a Sanofi company, and Isis Pharmaceuticals Inc., today announced that they have received the 2014 Partners in Progress Corporate Award from NORD in recognition of KYNAMRO (mipomersen sodium) injection. [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]