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Antisense is the non-coding strand in double-stranded DNA. The antisense strand serves as the template for mRNA synthesis.
Researchers gain new insight into how motor neurons in the brain die during ALS

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. [More]
Isis Pharmaceuticals begins ISIS-DMPK Rx clinical study in DM1 patients

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. [More]
ALS Association announces research funds to further understand genetic cause of ALS

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

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. [More]
Isis Pharmaceuticals CEO honored with SCRIP Lifetime Achievement Award

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. [More]
Researchers explore how to turn on the activity of paternal gene

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. [More]
Isis Pharmaceuticals announces initiation of ISIS-SMN Rx Phase 3 study in children with SMA

Isis Pharmaceuticals announces initiation of ISIS-SMN Rx Phase 3 study in children with SMA

Isis Pharmaceuticals, Inc. announced today the initiation of a pivotal Phase 3 study evaluating ISIS-SMNRx in approximately 120 non-ambulatory children with spinal muscular atrophy (SMA). [More]
Study gives a roadmap for future brain cancer vaccines

Study gives a roadmap for future brain cancer vaccines

Glioblastoma is the most common aggressive primary brain tumor, and despite advances in standard treatment, the median survival is about 15 months (compared to 4 months without treatment). [More]
Isis Pharmaceuticals, AstraZeneca partner to develop new delivery methods for antisense oligonucleotides

Isis Pharmaceuticals, AstraZeneca partner to develop new delivery methods for antisense oligonucleotides

AstraZeneca and Isis Pharmaceuticals Inc. today announced a strategic alliance to discover and develop novel delivery methods for antisense oligonucleotides. [More]
Isis receives $10 million from Biogen Idec to initiate IND-supporting studies of ISIS-BIIB3 Rx

Isis receives $10 million from Biogen Idec to initiate IND-supporting studies of ISIS-BIIB3 Rx

Isis Pharmaceuticals, Inc. announced today that it has earned a $10 million milestone payment from Biogen Idec related to the initiation of investigational new drug (IND)-supporting studies of ISIS-BIIB3Rx. ISIS-BIIB3Rx is a recently identified development candidate designed to inhibit an undisclosed target to treat a neurodegenerative disease. [More]
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]
New library sample preparation kits for Next Generation Sequencing announced by AMSBIO

New library sample preparation kits for Next Generation Sequencing announced by AMSBIO

AMSBIO announces RapidSeq – new convenient and cost-effective library sample preparation kits for Next Generation Sequencing (NGS) research and diagnostic work. [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]
Phase IIa clinical trial data of ATL1102 in MS patients published in journal Neurology

Phase IIa clinical trial data of ATL1102 in MS patients published in journal Neurology

Antisense Therapeutics is pleased to report the publication of previously generated Phase IIa clinical trial data on ATL1102 in the medical journal Neurology. [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]