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Targeting synthetic RNA to specific gene could help reduce severity of SMA

A team of University of Missouri researchers have found that targeting a synthetic molecule to a specific gene could help the severity of the disease Spinal Muscular Atrophy (SMA) - the leading genetic cause of infantile death in the world.

22 Nov 2011

Despite market constraints, the drug market for key hospital-treated infections will increase: Report

Decision Resources finds that the drug market for key hospital-treated infections that are of high interest to drug developers—most notably nosocomial pneumonia, bloodstream infections, skin and skin structure/surgical site infections and urinary tract infections—will increase from $3.3 billion in 2010 to just over $4 billion in 2020 in the United States, France, Germany, Italy, Spain, the United Kingdom and Japan.

21 Nov 2011

Akebia commences dosing in AKB-9778 Phase 1 study for diabetic macular edema and retinopathy

Akebia Therapeutics, Inc., a small molecule discovery and development company focused on anemia and vascular disorders, today announced that it has dosed the first subjects in a first-in-man Phase 1 study of AKB-9778, a first-in-class human protein tyrosine phosphatase beta (HPTPβ) inhibitor in development for diabetic macular edema (DME) and diabetic retinopathy (DR).

21 Nov 2011

Consistent data from three studies support plans for rindopepimut with ACT IV Phase 3 study

Celldex Therapeutics, Inc. today announced the presentation of mature overall survival (OS) data for ACT III, a multi-center, single arm, Phase 2 clinical trial of rindopepimut (CDX-110) in patients with newly diagnosed EGFRvIII-positive glioblastoma (GB).

21 Nov 2011

Positive preliminary results from Alnylam's ALN-TTR01 Phase I clinical trial for TTR-mediated amyloidosis

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today positive preliminary results from its Phase I clinical trial with ALN-TTR01, an RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR).

21 Nov 2011

Adeona to develop and commercialize DNA-based therapeutic for PAH using Intrexon technology

Adeona Pharmaceuticals, Inc., and the Human Therapeutics Division of Intrexon Corporation, announced today the formation of a global exclusive channel collaboration through which Adeona intends to develop and commercialize a DNA-based therapeutic using Intrexon's UltraVector platform and RheoSwitch Therapeutic System for the treatment of pulmonary arterial hypertension (PAH).

21 Nov 2011

CTI receives U.S. Patent Allowance for GlycoPolymer technology

Cell Therapeutics, Inc. (CTI) announced today that it has received a Notice of Allowance from the U.S. Patent and Trademark Office for a patent application directed to increasing the plasma half-life of therapeutic proteins and peptides by recombinantly conjugating them to a novel, non-immunogenic peptide repeat sequence.

21 Nov 2011

Shire, Shionogi enter agreement to co-develop and co-commercialize ADHD medicines in Japan

Shire plc, the global specialty biopharmaceutical company, announces that it has entered into an agreement with Shionogi & Co. Ltd. of Japan to co-develop and co-commercialize certain of Shire's Attention Deficit Hyperactivity Disorder (ADHD) medicines in Japan.

19 Nov 2011

Tikcro reports net loss of $1.0 million for third quarter 2011

Tikcro Technologies Ltd. today reported results for the third quarter and nine months ended September 30, 2011.

18 Nov 2011

Dendrimers can capture circulating tumor cells in blood by biomimicry

The removal of rare tumor cells circulating in the blood might be possible with the use of biomolecules bound to dendrimers, highly branched synthetic polymers, which could efficiently sift and capture the diseased cells, according to new research at the University of Illinois at Chicago.

18 Nov 2011

TNF inhibitors may minimally increase risk of serious infections

Biologic drugs have revolutionized treatment of autoimmune diseases during the past decade despite belief there is an increased risk for serious infections from using them. But new research from the University of Alabama at Birmingham Center for Education and Research on Therapeutics reveals that a class of biologics called tumor necrosis factor antagonists, or TNF inhibitors, may only minimally increase risk compared to more traditional therapies.

17 Nov 2011

CF Foundation, Genzyme announce new drug research agreement

Genzyme, a Sanofi company, and Cystic Fibrosis Foundation Therapeutics Inc., the nonprofit affiliate of the Cystic Fibrosis Foundation, today announced a research agreement to support the discovery of new drugs to treat people with the most common mutation found in patients with CF, Delta F508.

17 Nov 2011

Potential breakthrough in treating late-stage ovarian cancer

A potential breakthrough in treating late-stage ovarian cancer has come from University of Guelph researchers who have discovered a peptide that shrinks advanced tumours and improves survival rates for this deadly but often undetected disease.

17 Nov 2011

Mirna reports new preclinical data of miRNA-based therapeutic candidate for hepatocellular carcinoma

Mirna Therapeutics, Inc., a biotechnology company pioneering microRNA replacement therapy for cancer, announced the presentation today of new preclinical data on the development of a miRNA-based therapeutic candidate for primary liver cancer at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in San Francisco, CA.

16 Nov 2011

Aileron, Roche to expand Stapled Peptide drug collaboration

Aileron Therapeutics announced today that Roche will initiate a new program to expand its collaboration with Aileron to discover, develop and commercialize Stapled Peptide drugs.

16 Nov 2011

ZIOPHARM announces new preclinical data from two darinaparsin studies on solid tumors

ZIOPHARM Oncology, Inc., a drug development company employing small molecule and synthetic biology approaches to cancer therapy, announced today new preclinical data from two separate studies of darinaparsin, a novel organic arsenic, in various solid tumor models at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics, taking place November 12-16 in San Francisco.

16 Nov 2011

Researchers to begin drug development projects for rare and neglected diseases

Researchers will begin drug development projects for rare and neglected diseases that include potential treatments for a musculoskeletal disorder, a cognitive dysfunction disorder, a virus that affects the central nervous system of newborns, a parasitic worm infection, a form of muscular dystrophy and a rare lung disease.

16 Nov 2011

International consortium develops first-in-class adiponectin receptor agonist

A compound that mimics the effects of adiponectin, a beneficial protein that is produced by fat tissue in healthy weight individuals and may exhibit protective effects against cancer, cardiovascular disease, inflammatory conditions and insulin resistance has been developed by an international consortium lead by Dr. Eva Surmacz, Associate Professor in Biology (Adjunct) and director of the Obesity and Cancer Program at the Sbarro Institute for Cancer Research and Molecular Medicine at Temple University's College of Science and Technology.

16 Nov 2011

EMA grants orphan designation to AMT for hemophilia B gene therapy

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced today that the European Medicines Agency (EMA) has granted orphan designation for its gene therapy program for the treatment of hemophilia B. Orphan designation in the European Union provides several benefits including 10 years of market exclusivity from product launch and access to the central authorization procedure.

16 Nov 2011

EPO-secreting engineered vessels could reverse anemia

Patients who rely on recombinant, protein-based drugs must often endure frequent injections, often several times a week, or intravenous therapy. Researchers at Children's Hospital Boston demonstrate the possibility that blood vessels, made from genetically engineered cells, could secrete the drug on demand directly into the bloodstream.

15 Nov 2011

Therapeutics News and Research

Targeting synthetic RNA to specific gene could help reduce severity of SMA

Despite market constraints, the drug market for key hospital-treated infections will increase: Report

Akebia commences dosing in AKB-9778 Phase 1 study for diabetic macular edema and retinopathy

Consistent data from three studies support plans for rindopepimut with ACT IV Phase 3 study

Positive preliminary results from Alnylam's ALN-TTR01 Phase I clinical trial for TTR-mediated amyloidosis

Adeona to develop and commercialize DNA-based therapeutic for PAH using Intrexon technology

CTI receives U.S. Patent Allowance for GlycoPolymer technology

Shire, Shionogi enter agreement to co-develop and co-commercialize ADHD medicines in Japan

Tikcro reports net loss of $1.0 million for third quarter 2011

Dendrimers can capture circulating tumor cells in blood by biomimicry

TNF inhibitors may minimally increase risk of serious infections

CF Foundation, Genzyme announce new drug research agreement

Potential breakthrough in treating late-stage ovarian cancer

Mirna reports new preclinical data of miRNA-based therapeutic candidate for hepatocellular carcinoma

Aileron, Roche to expand Stapled Peptide drug collaboration

ZIOPHARM announces new preclinical data from two darinaparsin studies on solid tumors

Researchers to begin drug development projects for rare and neglected diseases

International consortium develops first-in-class adiponectin receptor agonist

EMA grants orphan designation to AMT for hemophilia B gene therapy

EPO-secreting engineered vessels could reverse anemia

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