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.

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

Translational Research issue focuses field of stem cell research, therapy

The use of stem cells for research and their possible application in the treatment of disease are hotly debated topics. In a special issue of Translational Research published this month an international group of medical experts presents an in-depth and balanced view of the rapidly evolving field of stem cell research and considers the potential of harnessing stem cells for therapy of human diseases including cardiovascular diseases, renal failure, neurologic disorders, gastrointestinal diseases, pulmonary diseases, neoplastic diseases, and type 1 diabetes mellitus.

10 Sep 2010

USPTO issues patent for Advaxis’ live Listeria HER2/neu immunotherapy

The U.S. Patent and Trademark Office has issued to Advaxis, Inc.,the live, attenuated Listeria monocytogenes (Listeria) immunotherapy company, patent number 7,784,729 titled "Methods and Compositions for Immunotherapy of Cancer," based upon patent application 10/949,667.

10 Sep 2010

SMA researchers receive grant for Spinal Muscular Atrophy research

Brian Kaspar, PhD, principal investigator in the Center for Gene Therapy at The Research Institute at Nationwide Children's Hospital, along with a team of Spinal Muscular Atrophy (SMA) researchers and clinicians, recently received a $250,000 grant for SMA research and clinic development from Sophia's Cure Foundation via the Pepsi Refresh Project.

9 Sep 2010

Advaxis receives USPTO patent for Listeria vaccine

The U.S. Patent and Trademark Office has approved Advaxis, Inc.,the live, attenuated Listeria monocytogenes (Listeria) immunotherapy company, intellectual property protection to patent application 11/223,945 - a live, attenuated Listeria vaccine that secretes novel fragments of the tumor antigen HER2/neu (human epidermal growth factor receptor 2), when using the company's proprietary listeriolysin O (LLO) fusion platform technology.

9 Sep 2010

Scientists gather to present research findings at ASHG 2010 Meeting

The world's top scientists and clinicians in the human genetics field will gather in Washington, D.C., to present their latest research findings at the 60th Annual Meeting of the American Society of Human Genetics, from Tuesday, November 2, through Saturday, November 6, 2010, at the Walter E. Washington Convention Center in D.C.

8 Sep 2010

Research on iPS cells can imply potential cell therapy for X-linked disorders

Female induced pluripotent stem (iPS) cells, reprogrammed from human skin cells into cells that have the embryonic-like potential to become any cell in the body, retain an inactive X chromosome, stem cell researchers at UCLA have found.

4 Sep 2010

UB symposium to discuss in silico molecular design, high-throughput screening to treat diseases

Twenty-first-century pharmaceutical breakthroughs require 21st-century drug discovery tools, such as computational or in silico molecular design and high-throughput screening of effective, new compounds. That's the theme of a University at Buffalo symposium to be held Sept. 11 on "Twenty-first Century Bioscience: In Silico Methods and High-Throughput Screening," which will feature a variety of cutting-edge advances in the field developed by researchers in Western New York and throughout the US.

3 Sep 2010