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.

Muscular Dystrophy Association awards $14.1M in new grants

The Muscular Dystrophy Association, which has invested almost $39 million in 2010 in worldwide research seeking treatments and cures for muscle diseases, today announced that it has awarded $14.1 million in new grants.

20 Aug 2010

MDA awards $14.1 million in new grants to advance treatments and cures for muscle diseases

18 Aug 2010

AMT's MAA for LPLD gene therapy product Glybera on schedule

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today announced that the Marketing Authorisation Application (MAA) for Glybera(R) remains on schedule following meetings with the European Medicine Agency (EMA) concerning the Day 120 List of Questions. The company is confident that Glybera(R), a gene therapy product for lipoprotein lipase deficiency (LPLD), remains on track for a regulatory decision by mid-2011.

18 Aug 2010

Life Technologies, CEVEC Pharmaceuticals sign licensing agreement to supply CAP-T expression kits

CEVEC Pharmaceuticals, the developer of a novel human protein expression system derived from amniocytes, announced today the signing of a co-exclusive worldwide licensing agreement with Life Technologies Corporation, a leading provider of innovative life science solutions based in Carlsbad, California. Under the terms of the agreement, Life Technologies and CEVEC will co-operate to offer optimized expression kits based on CEVEC's proprietary CAP-T cells to the global life science research community.

18 Aug 2010

Cryo-Cell International collaborates with MNSH to identify potential therapy for autoimmune diseases

Cryo-Cell International, Inc., one of the largest and most established leaders in stem cell innovation, today announced a research and development collaboration agreement with Monash University in Australia. The partnership will allow scientists from the University's Centre of Inflammatory Diseases to conduct pre-clinical studies using Cryo-Cell's proprietary C'elle menstrual stem cell technology (MenScs) to identify potential future therapies to treat autoimmune diseases such as multiple sclerosis.

18 Aug 2010

Non-viral gene therapy delays onset of eye disease

In one of only two studies of its kind, a study from researchers at Tufts University School of Medicine and the Sackler School of Graduate Biomedical Sciences at Tufts demonstrates that non-viral gene therapy can delay the onset of some forms of eye disease and preserve vision. The team developed nanoparticles to deliver therapeutic genes to the retina and found that treated mice temporarily retained more eyesight than controls.

17 Aug 2010

Scientists replace human gene to preserve photoreceptor function in retinal degeneration

The easy accessibility of the eye and the established link between specific genetic defects and ocular disorders offer hope for using gene therapy to provide long-term therapeutic benefit. Two reports in the current issue of Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc., describe the effective replacement of a human gene to preserve photoreceptor function in a mouse model of severe retinal degeneration. The articles are available free online.

16 Aug 2010

Neurologix second-quarter net loss increases to $4.5 million

Neurologix, Inc., a biotechnology company developing innovative gene therapies for the brain and central nervous system, today reported its financial results for the three and six months ended June 30, 2010.

13 Aug 2010

Tracking heart rate can provide important marker of health issues: Research

An elevated resting heart rate that develops or persists during follow-up is associated with a significantly increased risk of death, whether from heart disease or other causes, researchers from the Ronald O. Perelman Heart Institute at NewYork-Presbyterian Hospital/Weill Cornell Medical Center found studying outcomes in more than 9,000 patients.

13 Aug 2010

American Society of Hematology offers recommendations to propel collaborative research in regenerative medicine

The American Society of Hematology has developed specific recommendations to the scientific community and federal agencies to help propel collaborative research in regenerative medicine in order to make real strides in improving patient care. These recommendations were released today in Blood, ASH's premier scientific journal, in the article titled "Enhancing Research in Regenerative Medicine."

13 Aug 2010

Advaxis, Vibalogics to develop storage-stable formulation for live Listeria vaccines

Advaxis, Inc., the live, attenuated Listeria monocytogenes (Listeria) immunotherapy company and Vibalogics GmbH, a German biologics manufacturing company, have agreed to co-develop a room temperature stable, processing technology for live vaccines originally engineered for Advaxis.

12 Aug 2010

Venom-based nanoparticles improve gene therapy for fighting glioma

An ingredient in the venom of the "deathstalker" scorpion could help gene therapy become an effective treatment for brain cancer, scientists are reporting. The substance allows therapeutic genes - genes that treat disease - to reach more brain cancer cells than current approaches, according to the study in ACS Nano, a monthly journal.

12 Aug 2010

Spinal muscular atrophy may lead to cardiac dysfunction: Study

Along with skeletal muscles, it may be important to monitor heart function in patients with spinal muscular atrophy (SMA). These are the findings from a study conducted by Nationwide Children's Hospital and published online ahead of print in Human Molecular Genetics. This is the first study to report cardiac dysfunction in mouse models of SMA.

11 Aug 2010

Columbus hospital receives PPMD grant to conduct clinical testing for Duchenne

Parent Project Muscular Dystrophy, the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy (Duchenne), will award a $600,000 grant to Nationwide Children's Hospital in Columbus, Ohio to conduct clinical testing of a promising gene therapy technique for muscle disease.

10 Aug 2010

Neurobiologists discover potential cure for degenerative vision diseases

Neurobiologists funded by the Office of Naval Research have discovered a potential cure for degenerative vision diseases leading to terminal blindness.

5 Aug 2010

Vical second-quarter revenues decrease to $2.1 million

Vical Incorporated today reported financial results for the three months and six months ended June 30, 2010. Revenues decreased to $2.1 million for the second quarter of 2010 from $4.0 million for the second quarter of 2009, primarily as a result of a $1.5 million milestone payment in 2009 from Merck & Co., Inc., based on Merck's ongoing Phase 1 clinical-stage development of an investigational cancer vaccine.

3 Aug 2010

FDA lifts hold, allows Geron to proceed with Phase I clinical trial of GRNOPC1 in spinal cord injury

Geron Corporation announced today that the U.S. Food and Drug Administration (FDA) has notified the company that the clinical hold placed on Geron's Investigational New Drug (IND) application has been lifted and the company's Phase I clinical trial of GRNOPC1 in patients with acute spinal cord injury may proceed.

30 Jul 2010

Clontech Laboratories, TET Systems expand license agreement

Clontech Laboratories, Inc., a wholly-owned subsidiary of Takara Bio Inc., and TET Systems (TET), a privately-held German biotech company based in Heidelberg, today announced that they have signed an amendment to their existing license agreement. Under the expanded license agreement, Clontech obtains rights to develop, manufacture and commercialize novel Tet Systems products as the Tet-On® 3G™ Inducible Expression System.

26 Jul 2010

Valproic acid halts vision loss in patients with retinitis pigmentosa

Researchers at the University of Massachusetts Medical School believe they may have found a new treatment for retinitis pigmentosa, a severe neurodegenerative disease of the retina that ultimately results in blindness. One of the more common retinal degenerative diseases, RP is caused by the death of photoreceptor cells and affects 1 in 4,000 people in the United States. RP typically manifests in young adulthood as night blindness or a loss of peripheral vision and in many cases progresses to legal blindness by age 40.

22 Jul 2010

Encorium CRO acquires Progenitor

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 acquired Progenitor Holdings AG, a corporation organized in Switzerland and its wholly-owned subsidiaries organized in Mexico, Panama, Argentina, Chile, Switzerland, India and Hong Kong for cash and stock valued at EURO 1.7 million, plus earn-out consideration of cash and stock with a value of up to EURO 1.5 million.

19 Jul 2010