Gene Therapy News and Research

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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

New reference material to standardize gene therapy protocols in clinical trials

The introduction of a new, fully characterized viral vector for use as reference material to help standardize gene therapy protocols in research applications and human clinical trials is described in an article in Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc. (www.liebertpub.com). The article, which is published online ahead of print, is available free online at www.liebertpub.com/hum

7 Oct 2010

Immune reaction to dystrophin may contribute to muscle disease: Study

An immune reaction to dystrophin, the muscle protein that is defective in patients with Duchenne muscular dystrophy, may pose a new challenge to strengthening muscles of patients with this disease, suggests a new study appearing in the October 7, 2010, issue of The New England Journal of Medicine.

7 Oct 2010

Patients with muscular dystrophy mount immune response to dystophin protein prior to gene therapy: Study

A clinical trial designed to replace the genetic defect causing the most common form of muscular dystrophy has uncovered an unexpected aspect of the disease. The trial, based on therapy designed by scientists at the University of North Carolina at Chapel Hill School of Medicine, showed that some patients mount an immune response to the dystrophin protein even before they have received the gene therapy.

7 Oct 2010

Study demonstrates potential of nontoxic envelope proteins as an alternative to pseudotype lentiviral vectors

Therapeutic lentiviral vectors are emerging as vital tools for molecular medicine as evidenced by the growing number of clinical trials using these vector systems. From a basic research standpoint, lentiviral vectors are very intriguing substrates. On the one hand, the HIV-1 genome offers expanded cloning capacity and the capability to transduce nondividing cells such as hematopoietic stem cells (HSCs) and neurons.

4 Oct 2010

Encorium announces extension of expiration date for pending rights offering

Encorium Group, Inc. , a full service multinational clinical research organization (CRO) conducting studies in over 30 countries for many of the world's leading pharmaceutical and biotechnology companies, today announced that it has extended the expiration date for its pending rights offering from September 30, 2010 to October 15, 2010. The subscription rights may now be exercised at any time prior to 5:00 p.m. ET on Friday, October 15, 2010.

1 Oct 2010

NIH awards three grants to explore novel treatment strategies for muscular dystrophy

Three grants totaling more than $4.5 million, from agencies of the National Institutes of Health, will be used to explore novel treatment strategies for muscular dystrophy.

30 Sep 2010

CAP cells produce high virus titers upon influenza strain infection

CEVEC Pharmaceuticals, the developer of an extremely potent protein expression system based on human CAP cells, announced today that CAP cells produce high virus titers upon infection with influenza strains. The virus titers are as good as or even higher than with MDCK and Vero cells, host cells currently used for influenza vaccine production.

29 Sep 2010

Progenika receives CE Mark approval to market LPLchip in Europe

Progenika Biopharma S.A., a pioneer in the field of personalized medicine, announced today that it has obtained the CE Mark allowing the sale in Europe of its LPLchip(R), the first DNA chip to detect mutations in the lipoprotein lipase (LPL) gene.

27 Sep 2010

FDA designates AMT-080 gene therapy for Duchenne muscular dystrophy

Amsterdam Molecular Therapeutics (AMT) Holding N.V. a leader in the field of human gene therapy, announced today that the U.S. Food and Drug Administration (FDA) has designated AMT-080, a gene therapy for Duchenne muscular dystrophy (DMD) as an orphan drug.

24 Sep 2010

G-CSF compound may activate virus, increases risk of infection: Research

Research conducted at Oregon Health & Science University's Vaccine and Gene Therapy Institute may spur debate about the risks associated with administering a specific compound in some forms of bone-marrow transplantation. The research is published in the current edition of Cell Host and Microbe.

22 Sep 2010

NIDA's Avant-Garde Awards support novel approaches for treating tobacco and cocaine addiction

A potential immunotherapy, a new gene therapy, an enzyme inhibitor, and a compound originally isolated from a Chinese herb are among the latest approaches scientists are proposing to treat addiction.

22 Sep 2010

bluebird bio publishes LentiGlobin gene therapy clinical trial data results

bluebird bio an emerging leader in the development of innovative gene therapies for severe genetic disorders, today announced publication in the journal Nature of its promising Phase 1/2 data highlighting positive results of LentiGlobin gene therapy treatment in a young adult with severe beta-thalassemia, a blood disorder that is one of the most frequent inherited diseases.

16 Sep 2010

BCBC Consortium aims to develop cell-based therapy for insulin delivery

Oregon Health & Science University's Markus Grompe, M.D., will lead a multi-center team of scientists selected to participate in the prestigious Beta Cell Biology Consortium (BCBC). Funded by the National Institute of Diabetes and Digestive and Kidney Diseases, the consortium's long-term goal is to develop a cell-based therapy for insulin delivery in type 1 diabetes patients.

16 Sep 2010

Study finds GRK2 molecule promotes cell death after heart attack

A molecule known to be involved in progressive heart failure has now been shown to also lead to permanent damage after a heart attack, according to researchers at Thomas Jefferson University.

16 Sep 2010

AGTC receives grant to evaluate Phase II Human Clinical Trial for LCA retinal disease

Applied Genetic Technologies Corporation, a privately-held, clinical stage biotechnology company developing novel systems to deliver human therapeutics, announces that it has received a grant of $1 million from the Food and Drug Administration (FDA Orphan Drugs Program). The grant will fund a Phase II Human Clinical Trial evaluating the safety and efficacy of a treatment for Leber congenital amaurosis (LCA), a genetic retinal disease known to cause blindness at an early age.

14 Sep 2010

AskBio enters patent license agreement with Pfizer for HCV therapy

Asklepios BioPharmaceutical, Inc., a privately-owned clinical-stage biotechnology company, announced today it has entered into a patent license agreement with Pfizer, Inc. granting Pfizer access to certain proprietary gene-delivery platform technologies developed by AskBio, for use in developing novel therapies for patients with Hepatitis C virus (HCV).

14 Sep 2010

Myopia and Glaucoma genes identified

In global studies of more than 40,000 people from countries around the world, including Australia, researchers have found the genes responsible for two of the most common eye disorders - short-sightedness or myopia and glaucoma. The scientists believe this could open new diagnostic and management options.

13 Sep 2010

Heart Failure Society of America's 14th Annual Scientific Meeting convenes September 12, 2010

The Heart Failure Society of America (HFSA) 14th Annual Scientific Meeting convened Sunday, September 12, 2010 at the San Diego Convention Center in San Diego. This four-day meeting -- a forum for heart failure specialists to present research findings and advances in treatment -- will address emerging trends in research and new developments in the approach to treating patients with all stages of heart failure.

13 Sep 2010

Scientists discover gene associated with myopia

Myopia (nearsightedness) is the most common eye disorder in the world and becoming more common, yet little is known about its genetic underpinnings.Scientists at Duke University Medical Center, in conjunction with several other groups, have uncovered a gene associated with myopia in Caucasian people from several different regions, including Dutch, British and Australian subjects.

13 Sep 2010

Advaxis publishes research article on Her2 antigen for Listeria vaccine

Scientists at Advaxis, Inc.,the live, attenuated Listeria monocytogenes (Listeria) immunotherapy company, recently had their research using ADXS31-164, a live Listeria vaccine that targets Her2, published in a leading peer reviewed journal (Shahabi, et al 2010 Cancer Gene Therapy epublished ahead of print).

13 Sep 2010