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Innovators

The technologies developed for gene therapy was made thanks to independent and collaborative works done by scientists and researchers all over the US and the world.



Below you will find important gene therapy innovators

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Major Organizations

ASGCT

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The American Society of Gene and Cell Therapy, also known as the ASGCT, hopes to advance knowledge and education in order to lead the discovery for genetic and cellular therapy. By bringing together scientists, physicians, patient advocates, and other people, they hope to use genetic and cellular therapy to control and cure various human disease. It is a nonprofit organization, and is the largest medical organization dedicated to discovering new technologies and techniques for gene therapy. There are currently more than 2,000 members worldwide.

ESGCT

The ESGCT, or the European Society of Gene and Cell Therapy, is dedicated to the development of gene therapy, cell therapy, and the improvement of genetic vaccines. It is nonprofit and served as a professional adviser and regulatory body on gene therapy in Europe. It works to promote clinical research to further advance new technologies.

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ISCGT

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ISCGT stands for the International Society for Cell and Gene Therapy of Cancer. This society focuses on scientific collaboration with people around the world to develop the use of cellular and gene therapy for cancer. It works closely with not just national organizations, but also with local clinics.

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University Based Innovators

UC Davis Medical Center

Maintaining an annual budget of roughly $1 billion, UC Davis Medical Center operates in 33 counties, and care for more than 200,000 patients every year. Although it does not strictly focus on gene therapy research, it has collaborated and study multiple types of technologies for the benefit of human health. Recently it experimented with a high-tech drug designed to help sufferers of Duchenne, a muscular dystrophy disease which weakened skeletal muscles and delays development in sitting and walking. Working with a team consisting of pediatrics, cardiologist, radiologist, and researchers, they conducted an extensive study on a drug that utilizes oligonucleotide-mediated genetic therapy. 

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Dep. of Gene and Cell Medicine

Mount Sinai School of Medicine's Department of Gene and Cell Medicine in New York focuses on multiple aspects of gene therapy. They study the process of gene delivery, genetic incorporation  and virology. With multiple research laboratory, the Department of Gene and Cell Medicine is transforming and advancing clinical studies and research. For example, in 2010 Mount Sinai researchers developed a gene therapy procedure which enables the production of an enzyme that makes failing hearts pump more effectively.

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Program in Human Gene Therapy

The Program in Human Gene Therapy at Stanford University aims to develop gene transfer technologies in order to treat genetic and acquired diseases. Focusing on a variety of viral and nonviral vectors, this program uses major disease models like hemophilia, hepatitis C and B viral infections, and diabetes. In addition, the program also focuses on small RNAs and their roles in mammalian gene regulation.

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Minnesota University

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The Molecular and Cellular Therapeutics Program in Minnesota University follows complex and detailed regulations and requirements needed for cGMP production of cell and tissues based products, monoclonal antibodies, and other pharmaceutical ingredients. Through these products, the Molecular and Cellular Therapeutics Program hopes to shorten developmental timelines and change current research into active medicine.

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Private Innovating Companies

BioCardia , Inc

BioCardia, Inc developed a delivery catheter system called Helix Biotherapeutic Delivery, which enables delivery of cell and gene based therapies to treat heart failure, myocardial infarction, ischemiam, and cardiac conduction disorders. It is the leading biotherapeutic delivery catheter system for cardiovascular regenerative medicine.

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Transgene S.A.

Transgene is a biopharmaceutical company which designs and develops products to treat chronically infectious diseases and cancers. It has designed a proprietary gene transfer technology, and is a major researcher in immunotherapy. It currently has four products in clinical development and in phase II trials.

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MediGene, Inc

Medigene, Inc. focuses on novel drugs for use in cancer, autoimmune diseases, and skin diseases. It is the first biotech company in Germany to generate profit from products on the market. Medigene currently has two potential candidates which are in clinical development. In addition to these drugs, Medigene is developing a new vaccine technology to commercialized.

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Applied Tissue Technologies LLC

Applied Tissue Technologies LLC designs, develops, and sells wound healing technology. Founded in 2000, ATT developed XPANSION Skin Grafting Instrument Kit, which is used to heal acute and chronic wounds which fails to regenerate and heal. In late 2012, ATT will be targeting the world international markets with its Skin Grafting Kit.

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OrphageniX

OrphageniX is researching and utilizing proprietary single stranded oligonucleotides to treat orphan diseases, which are rare and extremely dangerous. The patented singled stranded oligonucleotides are used as drug candidates for several incurable genetic diseases.

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