Recent News from Gene and Cell Therapy Field

Everybody acknowledges that we are at the verge of entering a new era in medicine full of scientific discoveries and breakthroughs. Gene and Cell Therapy surely will alter the way medicine is practiced. Below are just few examples of what Gene and Cell Therapy can do for your patient. More to come in the future.


Glybera® (alipogene tiparvovec) is a gene therapy that is designed to restore the LPL enzyme activity required to enable the processing, or clearance, of fat-carrying chylomicron particles formed in the intestine after a fat-containing meal. The product consists of an engineered copy of the human LPL gene packaged with a tissue-specific promoter in a non-replicating AAV1 vector, which has a particular affinity for muscle cells. In order to improve activity, uniQure uses a naturally occurring variant of the LPL gene that has higher enzyme activity than the normal version of the gene that encodes the protein. The company produces Glybera using its insect cell-based manufacturing process. Clinicians administer Glybera in a one-time series of up to 60 intramuscular injections in the legs. The patient is administered spinal anesthesia or deep sedation during the procedure. In addition, an immunosuppressive regimen is recommended from three days prior to and for 12 weeks following Glybera administration.

Glybera Regulatory Status:

In October 2012, the European Commission granted marketing authorization for Glybera® under exceptional circumstances as a treatment for adult patients diagnosed with familial lipoprotein lipase deficiency (LPLD) confirmed by genetic testing, and suffering from severe or multiple pancreatitis attacks despite dietary fat restrictions.

Two severe genetic diseases are successfully treated with blood stem cells gene therapy using vectors derived from HIV

The AIDS virus can be used to treat two severe hereditary diseases. After years of study in the laboratory to engineer the virus and design the gene therapies, six children from all over the world, now three years after the treatment, are well and show significant benefits. The announcement was made in two studies published today in Science by researchers at the San Raffaele Telethon Institute for Gene Therapy (TIGET) in Milan, Italy, led by Luigi Naldini, demonstrating that hematopoietic stem cell gene therapy works against two severe genetic diseases, metachromatic leukodystrophy and Wiskott-Aldrich syndrome.

Both trials, which involved a team of over 70 people including researchers and clinicians, began in the spring of 2010, and called for the participation of 16 patients in total, 6 suffering from Wiskott-Aldrich syndrome and 10 from metachromatic leukodystrophy. The results published in Science refer only to the first 6 patients (three from each study), i.e. those for whom sufficient time has passed after administration of the therapy to allow scientists to draw the first significant conclusions regarding its safety and efficacy.

In 2010, Telethon Foundation and San Raffaele Institute entered into a strategic alliance with GlaxoSmithKline to develop and commercialize ex vivo stem cell gene therapies developed at TIGET. Under the terms of the agreement, GSK has an exclusive option to further develop the treatments for metachromatic leukodystrophy and Wiskott-Aldrich Syndrome reported in these studies.

Gene Therapy and Stem Cells Unite

Two of the holy grails of medicine - stem cell technology and precision gene therapy - have been united for the first time in humans, say scientists.

It means patients with a genetic disease could, one day, be treated with their own cells.

Gene Therapy and Stem CellsRead more on the story from BBC News By James Gallagher

The History and Promise of Gene Therapy by James M. Wilson

The potential of treating or preventing disease by modifying the expression of one's genes has captured the imagination of the scientific community ever since the discovery of DNA.

This therapeutic concept, called gene therapy, emerged as a very literal clinical application of modern day molecular biology. While the concept may seem simple, its translation into effective therapies has been challenging. The events that led up to this impending revolution are summarized below.

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Gene therapy saves a man from losing his sight

Researchers in Oxford have treated a man with an advanced gene therapy technique to prevent him from losing his sight.

It is the first time that anyone has tried to correct a genetic defect in the light-sensing cells that line the back of the eye.

The president of the Academy of Medical Sciences said the widespread use of gene therapy of this treatment will be soon be possible.

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Molecular diagnostics for congenital color blindness

Genevolve Vision Diagnostics developed a noninvasive molecular diagnostic for all major forms of congenital color blindness, for use by ophthalmologists and optometrists.

The test, marketed under the Eyedox™ brand, involves using a noninvasive buccal epithelial cell collector to pick up skin cells sloughed daily from the lining of the mouth.The DNA goes to a CLIA-certified laboratory where the color genes are identified and classified for inherited color vision deficiency, and a treatment plan and family history are developed.

While gene therapy has been shown to cure some forms of congenital blindness, better diagnostics will be critical for identifying patients for current and future gene therapies.

Cystic Fibrosis Trust

The U.K. CF Gene Therapy Consortium, funded by the Cystic Fibrosis Trust, seeks to raise €6 million for a gene therapy trial.

As of October 6, supporters raised €700,000 toward funding the multidose clinical trial, which calls for more than 100 people with cystic fibrosis in Edinburgh and London to be given either a healthy gene, the gene transfer agent, and the promoter or a placebo, once a month for a year.

If patient lung function shows improvement, the consortium has said it will pursue a pharmaceutical partner to take the product into a Phase III clinical trial starting in 2015.

Advanced chronic lymphocytic leukemia

University of Pennsylvania researchers reported sustained remissions of up to a year among a small group of advanced chronic lymphocytic leukemia patients treated with genetically engineered versions of their own T cells.

Carl June, M.D., led a team from the Abramson Cancer Center and Perelman School of Medicine in removing patients' cells, modifying them in UPenn's vaccine production facility, then infusing the new cells back into patients' bodies after chemotherapy.

The findings, published simultaneously in the New England Journal of Medicine and Science Translational Medicine, are the first demonstration of gene transfer therapy to create "serial killer" T cells aimed at cancerous tumors, a possible roadmap for treating other cancers.

Stem-cell-like T cell

U.S. and British researchers identified a highly undifferentiated stem-cell-like T cell within a patient's immune system that can continually refresh itself for long periods of time, possibly permanently.

Writing in Nature Medicine, the researchers from NCI, NIAID, NIH, UPenn, and Cardiff University School of Medicine discussed how they isolated a tiny fraction of highly undifferentiated T cells showing the first changes in gene expression that occur when T cells "remember" antigens they have seen previously, and prepare to react quickly.

NCI's Nicholas P. Restifo, M.D., and his team hope to regenerate younger T cells from older ones by manipulating certain genes.

FDA advisory panel

An FDA advisory panel identified three challenges to development of cellular and gene therapies for retinal disorders in adult and pediatric populations.

FDA's Cellular, Tissue, and Gene Therapies Advisory Committee pinpointed the challenges as: 1)Selection of efficacy endpoints for clinical trials, especially for trials intended to study rare retinal disorders and disorders in very young children; 2)Treatment of the contralateral eye and repeat administration of the product; and 3)Identifying methods to confirm accurate delivery of the intended dose into the target site, in both preclinical and clinical studies.

No cell or gene therapy products are licensed for ophthalmic indications in the U.S., but several such products are in development.

Celebrating 10 years after gene therapy of SCID

Christopher Reid and his family are celebrating 10 years since he became the first child in England to receive gene replacement therapy for severe combined immuno-deficiency (SCID), a genetic condition that meant he had no natural immune system and was forced to live in a protective "bubble".

He was diagnosed with SCID at eight months of age, and given three months to live. But in December 2001, Christopher underwent an operation in which an artificially created gene was mixed with his bone marrow, and injected into his body.

Gene therapy is now considered a cure for SCID, as reported in Science Translational Medicine in August.

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Human Gene Therapy Methods

The field of gene therapy is evolving and and holds great promise for its applications in treating human disease. Human Gene Therapy Journal, the premier in the field, has launched Human Gene Therapy Methods to complement the flagship publication.

Gene and Cell Therapy

The purpose of Turkish Society of Gene and Cell Therapy is to enhance and foster Gene and Cell Therapy Research in Turkey

Turkish Society of Gene and Cell Therapy

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Gene Therapy Helps Blind Children See

eye gene therapy

A single injection of DNA into the eyes of four children born with a blindness-causing disease has given them enough vision to walk without help.

The study, published today, confirms that if patients with this disease are given gene therapy early in life, the results can be dramatic.

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Terminating 'Serial Killers' Makes Cell Therapy Safer:

Texas Medical Center

Cancer treatments with stem cell transplantation and specially modified T-cells can be made safer by using a new termination system that almost immediately controls the therapy if things go wrong, said researchers.

Dr. Malcolm Brenner, director of the Center for Cell and Gene Therapy, and his colleagues have been developing special anti-tumor cells called T-cells that harness the body's immune system to fight cancer.

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Bubble boy disease

Jack Crick

The gene and cell therapy has given 7 years old Jack Crick his life back.

Researchers reported results of a trial that show that gene therapy was succesful in 14 out of the 16 UK children they treated, allowing them to recover and live normal lives. Click on the images to read more on the story.

bubble boy

Gene therapy success for haemophilia

Queen Victoria

Just one injection could be enough to mean people with haemophilia B no longer need medication, according to an early study in the UK and the US.

Doctors said the gene therapy was "potentially life-changing". Other researchers have described it as a "truly a landmark study."

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