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.

Researchers find new way to prevent blindness caused by retinitis pigmentosa

Researchers at the University of Oklahoma Health Sciences Center have found a way to use a radical new type of gene therapy to prevent blindness caused by retinitis pigmentosa, giving hope to the estimated 100,000 Americans who suffer from this debilitating disease.

14 Jun 2010

UK scientists discover cancer-blocking activity in Down's syndrome chromosome

Cancer Research UK scientists have discovered cancer-blocking activity for genes carried on chromosome 21 - an extra copy of which is carried by people with Down's syndrome, reveals research published in Nature.

11 Jun 2010

Research may lead to better treatments for depression and substance abuse

In what has been hailed as a breakthrough, scientists from Columbia University Medical Center and Weill Cornell Medical College have outlined the molecular mechanism of membrane transport. The research shows how a protein transforms its shape to transport substances across the cell membrane in order to regulate transmission of the brain's messages across the synaptic gap from one neuron to another.

11 Jun 2010

PPMD to fund two promising research initiatives in Duchenne muscular dystrophy

Patricia A. Furlong, Founding President and CEO of Parent Project Muscular Dystrophy (PPMD), the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy (Duchenne), announced today that PPMD will be funding two critical, promising research initiatives in Duchenne.

11 Jun 2010

Novel gene replacement therapy holds promise for Spinal Muscular Atrophy

Children who suffer from the devastating disease Spinal Muscular Atrophy are set to benefit from a new breakthrough in therapy developments by researchers at the University of Sheffield.

10 Jun 2010

Takara Bio to distribute NuGEN's solutions in Japan, South Korea, China

NuGEN, the provider of innovative RNA and DNA sample preparation technologies that enable breakthrough genomic analysis, today announced it has signed a non-exclusive agreement with Takara Bio to distribute the full spectrum of NuGEN's next-generation sequencing, microarray and q-PCR nucleic acid amplification and sample preparation products in Japan, South Korea and China. In addition, Takara is authorized to offer services using NuGEN products in the same territory.

9 Jun 2010

SERCA2a gene therapy proves effective in people with advanced heart failure: Researchers

Researchers at Mount Sinai School of Medicine have developed a new gene therapy that is safe and effective in reversing advanced heart failure. SERCA2a (produced as MYDICAR-) is a gene therapy designed to stimulate production of an enzyme that enables the failing heart to pump more effectively.

4 Jun 2010

Sticky gel composed of MPEG-pDHA may help seal wounds created by surgery: Study

A compound found in sunless tanning spray may help to heal wounds following surgery, according to new results published by plastic surgeons from NewYork-Presbyterian Hospital/Weill Cornell Medical Center in New York City and biomedical engineers at Cornell University in Ithaca, N.Y., where the novel compound was developed.

1 Jun 2010

Celladon announces 6-month data from Phase 2 clinical trial of MYDICAR in advanced heart failure patients

Celladon Corp., a biopharmaceutical company focused on the discovery and development of innovative treatments for cardiovascular diseases, today announced that six-month data from its Phase 2 clinical trial of MYDICAR® show improvements in clinical outcomes and disease markers in advanced heart failure patients treated with the genetically targeted enzyme replacement therapy.

31 May 2010

Targeted Genetics's partner Celladon presents Phase II trial data with MYDICAR at Heart Failure Congress 2010

Targeted Genetics Corporation announced that its partner, Celladon Corporation, presented full data today that show the first Phase II trial with MYDICAR® for the treatment of advanced heart failure has met its primary safety and efficacy endpoints, as prospectively defined in the Phase II trial protocol.

31 May 2010

Hebrew Union College honors Dr. Dean Lorich with 2010 Roger E. Joseph Prize for humanitarian efforts

Dr. Dean Lorich was honored with the 2010 Roger E. Joseph Prize by Hebrew Union College for his humanitarian efforts in treating victims of the recent Haiti earthquake and in Landstuhl, Germany treating soldiers injured in Iraq and in Afghanistan. The award recognizes individuals making lasting contributions to the causes of human rights.

29 May 2010

SonoSite signs agreement to acquire privately held Visualsonics

SonoSite, Inc., the world leader and specialist in bedside and point-of-care ultrasound, today announced that it has signed an agreement to acquire privately held Visualsonics, a Toronto-based company focused on ultra high-frequency micro-ultrasound technology. The aggregate transaction value will be approximately $71 million net of cash and debt.

27 May 2010

Weill Cornell Medical College commences construction of 18-story, $650 million Medical Research Building

Weill Cornell Medical College today celebrated the start of construction on its new Medical Research Building, a state-of-the-art facility that will more than double the institution's existing research space. The 18-story, $650 million building is the centerpiece of Weill Cornell's $1.3 billion Discoveries that Make a Difference Campaign, the country's largest for a medical college. A total of $1 billion has been raised toward this goal in a record time of less than four years.

27 May 2010

Nottingham scientists given green light to test new vaccine for malignant melanoma

Nottingham scientists have been given the green light to test a vaccine which they hope could reverse, and even cure malignant melanoma, the most deadly type of skin cancer.

27 May 2010

Report: 235 new medicines in development to treat diabetes

A record 235 new medicines to treat diabetes, one of the fastest-growing diseases in America, are being developed by America's pharmaceutical research and biotechnology companies, according to a national report unveiled today in Charleston.

26 May 2010

CEVEC, Pevion Biotech sign license option agreement for transient CAP-T cell expression technology

CEVEC Pharmaceuticals, the German developer of a novel human expression system derived from amniocytes, announced today the signing of an exclusive license option agreement with Pevion Biotech, the Swiss vaccine company. The agreement enables Pevion Biotech to exclusively use CEVEC's transient CAP-T™ cell expression technology to manufacture and commercialize a protein antigen for vaccination against a widespread human infectious disease.

26 May 2010

QIAGEN acquires rights to 70 molecular food safety tests developed by Institute for Product Quality

QIAGEN today announced that it has entered into an agreement with the Institute for Product Quality (ifp), pursuant to which QIAGEN has acquired all rights to 70 molecular food safety tests developed by the Berlin-based specialized laboratory center for food analysis.

From QIAGEN Manchester Limited 26 May 2010

t2c001 product obtains certificate under EMA regulations for ATMPs

t2cure GmbH, a biopharmaceutical company developing and marketing stem cell-based regenerative therapies, announced today that the quality of its lead product t2c001 has been certified by the European Medicines Agency.

25 May 2010

Sangamo's ZFP Therapeutics technology highlighted at ASGCT Annual Meeting

Sangamo BioSciences, Inc. announced today that data from research and preclinical programs focused on the development of zinc finger DNA-binding protein Therapeutics were described in fifteen presentations given by Sangamo scientists and collaborators at the 13th Annual Meeting of the American Society of Gene and Cell Therapy.

24 May 2010

New therapy program serves as foundation for ongoing clinical development

The researchers of the University of Helsinki, and Oncos Therapeutics, the biotech company developing new cancer therapeutics based on the next generation oncolytic viruses, published initial results from their Advanced Therapy Access Program in Cancer Research.

21 May 2010

Gene Therapy News and Research

Further Reading

Researchers find new way to prevent blindness caused by retinitis pigmentosa

UK scientists discover cancer-blocking activity in Down's syndrome chromosome

Research may lead to better treatments for depression and substance abuse

PPMD to fund two promising research initiatives in Duchenne muscular dystrophy

Novel gene replacement therapy holds promise for Spinal Muscular Atrophy

Takara Bio to distribute NuGEN's solutions in Japan, South Korea, China

SERCA2a gene therapy proves effective in people with advanced heart failure: Researchers

Sticky gel composed of MPEG-pDHA may help seal wounds created by surgery: Study

Celladon announces 6-month data from Phase 2 clinical trial of MYDICAR in advanced heart failure patients

Targeted Genetics's partner Celladon presents Phase II trial data with MYDICAR at Heart Failure Congress 2010

Hebrew Union College honors Dr. Dean Lorich with 2010 Roger E. Joseph Prize for humanitarian efforts

SonoSite signs agreement to acquire privately held Visualsonics

Weill Cornell Medical College commences construction of 18-story, $650 million Medical Research Building

Nottingham scientists given green light to test new vaccine for malignant melanoma

Report: 235 new medicines in development to treat diabetes

CEVEC, Pevion Biotech sign license option agreement for transient CAP-T cell expression technology

QIAGEN acquires rights to 70 molecular food safety tests developed by Institute for Product Quality

t2c001 product obtains certificate under EMA regulations for ATMPs

Sangamo's ZFP Therapeutics technology highlighted at ASGCT Annual Meeting

New therapy program serves as foundation for ongoing clinical development

Event guide: 9th Gene Therapy Development Summit 2026 eBook

Evaluating new approaches for rare disease genomics

Expanding access to NMR with the Fourier 80 Duo

Advancing Intracellular Delivery with LumiPore®

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