1. Gene Therapy

Category Archives: Gene Therapy

Global Gene Therapy Market: Analysis, Size, Share, Growth, Trends and Forecast 2013 – 2019

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Albany, New York (PRWEB) May 22, 2014

Gene therapy involves use of DNA as a pharmaceutical agent to treat diseases. It is one of the most important developments in the field of medicine that has potential to treat various lethal diseases such as HIV, cancer and cystic fibrosis. In the long run, biotechnology and clinical trial industries will benefit from developments in gene therapy and provide potential treatment solutions for various incurable diseases.

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In the present scenario, various pharmaceutical companies are using clinical data to validate the concept of gene therapy. Moreover, many venture capital investors are also showing their interest in gene therapy, and are investing heavily in its development. However, gene therapy is highly dependent on the regulatory approvals and most of the products are currently in clinical trial phase. Most of these gene therapy products are for cancer and cardiovascular diseases, and are in Phase III/ Phase II of clinical trials.

In addition, growing popularity of DNA vaccines boost advances in gene therapy and is likely to be practiced in clinics in the near future, with a number of therapy programs now in phase II/III trials, showing promising results.

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Some of the major players operating in the market are AnGes MG, BioSante Pharmaceuticals, GenVec, Genzyme Corporation, Oxford BioMedica, Transgene, Urigen Pharmaceuticals and Vical.

This research report analyzes this market depending on its market segments, major geographies, and current market trends. Geographies analyzed under this research report include:

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This report is a complete study of current trends in the market, industry growth drivers, and restraints. It provides market projections for the coming years. It includes analysis of recent developments in technology, Porters five force model analysis and detailed profiles of top industry players. The report also includes a review of micro and macro factors essential for the existing market players and new entrants along with detailed value chain analysis.

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Global Gene Therapy Market: Analysis, Size, Share, Growth, Trends and Forecast 2013 - 2019

Gene therapy hits blood cancers hard

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In one of the biggest advances against leukemia and other blood cancers in many years, doctors are reporting unprecedented success by using gene therapy to transform patients blood cells into soldiers that seek and destroy cancer.

A few patients with one type of leukemia were given this one-time, experimental therapy several years ago, and some remain cancer-free today. Now, at least six research groups have treated more than 120 patients with many types of blood and bone marrow cancers, with stunning results.

Its really exciting, said Dr. Janis Abkowitz, blood diseases chief at the University of Washington in Seattle and president of the American Society of Hematology. You can take a cell that belongs to a patient and engineer it to be an attack cell.

In one study, all five adults and 19 of 22 children with acute lymphocytic leukemia, commonly known as ALL, had a complete remission, meaning no cancer could be found after treatment, although a few have relapsed since then.

These were gravely ill patients out of options. Some had tried multiple bone marrow transplants and up to 10 types of chemotherapy or other treatments.

Cancer was so advanced in Emily Whitehead, now 8, of Philipsburg, Pa., that doctors said her major organs would fail within days. She was the first child given the gene therapy; now almost two years later, she shows no sign of cancer.

The regimen also can be used to treat myeloma, lymphoma and chronic lymphocytic leukemia, commonly known as CLL.

This has the potential to become the first gene therapy approved in the United States and the first for cancer worldwide, doctors said. Only one gene therapy is approved in Europe, for a rare metabolic disease.

The treatment involves filtering patients blood to remove millions of white blood cells called T-cells, altering them in the lab to contain a gene that targets cancer, and returning them to the patient in infusions over three days.

What we are giving essentially is a living drug permanently altered cells that multiply in the body into an army to fight the cancer, said Dr. David Porter, a University of Pennsylvania scientist who led one study.

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Gene therapy hits blood cancers hard

Novel RNAi therapy silences mutated Huntington's disease gene and reduces symptoms

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PUBLIC RELEASE DATE:

21-May-2014

Contact: Kathryn Ruehle kruehle@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, May 21, 2014A targeted gene silencing strategy blocks production of the dysfunctional huntingtin (Htt) protein, the cause of Huntington's disease, a fatal, inherited neurodegenerative disorder. The effectiveness of this RNA interference (RNAi) approach in reducing levels of mutant Htt protein and disease symptoms in a mouse model of the disease is described in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Human Gene Therapy website.

Lisa Stanek and coauthors from Genzyme (Framingham, MA) used an adeno-associated viral (AAV) vector to deliver a targeted nucleic acid sequence called a small interfering RNA (siRNA) into the cells of affected mice. The siRNA selectively binds to the mutated gene, blocking disease-causing Htt production. The authors present data demonstrating the ability to deliver the therapeutic RNAi into the cells, reduce mutant Htt levels, and impact behavioral deficits in the mice without causing any noticeable neurotoxicity, in their article "Silencing Mutant Huntingtin by Adeno-Associated Virus-Mediated RNA Interference Ameliorates Disease Manifestations in the YAC128 Mouse Model of Huntington's Disease."

"The Genzyme group uses state-of-the-art delivery technology and a gene silencing approach to generate very promising preclinical data for Huntington's disease," says James M. Wilson, MD, PhD, Editor-in-Chief of Human Gene Therapy, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia.

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About the Journal

Human Gene Therapy, the official journal of the European Society of Gene and Cell Therapy, British Society for Gene and Cell Therapy, French Society of Cell and Gene Therapy, German Society of Gene Therapy, and five other gene therapy societies, is an authoritative peer-reviewed journal published monthly in print and online. Human Gene Therapy presents reports on the transfer and expression of genes in mammals, including humans. Related topics include improvements in vector development, delivery systems, and animal models, particularly in the areas of cancer, heart disease, viral disease, genetic disease, and neurological disease, as well as ethical, legal, and regulatory issues related to the gene transfer in humans. Its sister journals, Human Gene Therapy Methods, published bimonthly, focuses on the application of gene therapy to product testing and development, and Human Gene Therapy Clinical Development, published quarterly, features data relevant to the regulatory review and commercial development of cell and gene therapy products. Tables of content for all three publications and a free sample issue may be viewed on the Human Gene Therapy website.

About the Publisher

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Novel RNAi therapy silences mutated Huntington's disease gene and reduces symptoms

Nanoinjector Device: New Gene Therapy Advance from BYU Microbiologists and Engineers – Video

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Nanoinjector Device: New Gene Therapy Advance from BYU Microbiologists and Engineers
BYU researchers create tiny nano-device in newest gene therapy advance: Nanoinjector is used to transfer genes and DNA to new cells The ability to transfer a gene or DNA sequence from one animal...

By: BYU

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Nanoinjector Device: New Gene Therapy Advance from BYU Microbiologists and Engineers - Video

Spark Therapeutics to open headquarters in West Philadelphia

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Spark Therapeutics, a gene therapy medical company, this week signed an agreement for a headquarters in West Philadelphia. Spark Therapeutics, spun out of Children's Hospital of Philadelphia in October with $50 million in capital, will build out a 28,000-square-foot facility at 3737 Market St. to house its business operations, clinical research and development, and manufacturing.

Jeffrey D. Marrazzo, cofounder and chief executive, said the new facility "will support the continued expansion of our team and expand our manufacturing capacity to support our clinical development and commercial plans."

Spark anticipates moving into its new headquarters and expanding to 50 full-time employees by the end of 2014. Spark is preparing to complete clinical development of its lead, Phase 3 clinical program to address inherited retinal dystrophies caused by mutations in the RPE65 gene.

- Erin Arvedlund

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Spark Therapeutics to open headquarters in West Philadelphia

Employment of gene therapy in depression (psychiatric disorder) – Video

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Employment of gene therapy in depression (psychiatric disorder)
A study in 2010 discovered the gene responsible for causing symptoms of major depression. The discovery of this gene makes it possible to employ gene therapy...

By: EMPLOYMENT OF GENE THERAPY IN PSYCHIATRIC DISORDER

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Employment of gene therapy in depression (psychiatric disorder) - Video

Gene and Cell Therapy for Thrombosis, AIDS and Inherited Disorders

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Washington, DC (PRWEB) May 05, 2014

There will be a Media Event in Washington, DC, on Wednesday, May 21, 2014, from 10:45 am 12:15 pm in Wilson A of the Marriott Wardman Park Hotel, which is sponsored by the American Society of Gene & Cell Therapy (ASGCT).

The event will profile exciting clinical trial results in patients suffering from serious and often fatal diseases such as human immunodeficiency virus (HIV), Inherited Immunodeficiencies, Hemophilia B, and complications from Thrombosis. All registered media will have the opportunity for personal, one-on-one questions with academic and industry leaders in the field, including:

Dr. Bruce L. Levine of the University of Pennsylvania is developing a gene and cell therapy strategy to achieve a functional cure for HIV infection by genome editing of cells, and results from the first-in-human trial of this novel treatment strategy will be presented.

Professor Adrian Thrasher of the University College of Londons Institute of Child Health will discuss the impressive results of recent advances in gene therapy technology to treat inherited immunodeficiencies in patients, many of whom are children.

Researchers at St. Jude Children's Research Hospital have successfully treated Hemophilia patients with gene therapy that led to their disease-free living for several years. Dr. Andrew Davidoff will provide the latest update on these exciting clinical results.

Thrombosis, or the formation of blood clots inside a blood vessel, remains the major cause of death and disability in the western world. Dr. Bruce Sullenger of Duke University Medical Center will describe the recent development of gene and cell therapy strategies to control thrombosis that is currently in a Phase 3 clinical trial.

The media event will take place in Wilson A in the Marriott Wardman Park Hotel.

Members of the media are welcome to conduct individual interviews with each speaker following the presentation, and will receive complimentary full-access registration to the ASGCT 17th Annual Meeting. Representatives who wish to attend may contact ASGCT directly at 414.278.1341 or mdean(at)asgct(dot)org.

The American Society of Gene & Cell Therapy (ASGCT) is a professional nonprofit medical and scientific organization dedicated to the understanding, development and application of genetic and cellular therapies and the promotion of professional and public education in the field. For more information on ASGCT, visit its website, http://www.asgct.org.

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Gene and Cell Therapy for Thrombosis, AIDS and Inherited Disorders


  1. Gene Therapy