| Directory > Science > Biology > Genetics > Eukaryotic > Animal > Mammal > Human > Gene Therapy Murdoch Children's Research Institute - Cell and Gene Therapy
The CAGT group of MCRI focuses on gene therapy research for neurological, haematological and liver
diseases affecting babies, children and adolescents.
http://murdoch.rch.unimelb.edu.au/pages/research/research.asp?P=oview&G=10&T=Lab
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Goldyne Savad Institute of Gene Therapy
Aims to enhance the development of safe and effective gene therapy by supporting research projects,
from vector development and construction to production of biologicals for human use and their
application in well designed GCP clinical trials.
http://english.hadassah.org.il/departments/gen_ther/
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Targeted Genetics Corporation
Develops gene delivery technologies and therapeutic products based on gene delivery.
http://www.targen.com
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Jesse's Journey - The Foundation for Gene and Cell Therapy
A registered charitable organization raising funds for research in gene and cell based therapies
focusing on Duchenne muscular dystrophy and other neuromuscular diseases.
http://www.jessesjourney.com
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National Gene Vector Laboratories (NGVL)
An interactive group of academic production and pharmacology/toxicology laboratories whose primary
goal is to provide eligible investigators with clinical grade vectors for phase I/II gene therapy
clinical trials and to provide support for relevant pharmacology/toxicology studies leading up to
clinical gene transfer protocols.
http://www.ngvl.org
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Polyplus Transfection
Dedicated to the development, manufacturing and marketing of modular transfection reagents.
Expertise includes in vitro and in vivo gene, oligonucleotide and siRNA delivery, transient and
stable cell transfection, automation and protein production in animal cells.
http://www.polyplus-transfection.com
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Henogen
A GMP biomanufacturing company located in Belgium, able to produce, purify and aseptically fill
clinical batches of therapeutic proteins, monoclonal antibodies, plasmid DNA, vaccinal viruses or
viral vectors for gene therapy.
http://www.henogen.com/
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Gene Therapy - Human Genome Project
Definition, hurdles, ethical issues, and links to more resources on gene therapy provided by the
Oak Ridge National Laboratory.
http://www.ornl.gov/hgmis/medicine/genetherapy.html
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Cobra BioManufacturing
Developing gene therapies for cancer and DNA vaccines to stimulate cytotoxic T cell (CTL)
responses. Also offers custom GMP manufacturing for plasimd DNA, viral and non-viral vectors.
http://www.cobrabiomanufacturing.com/
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The Children's Hospital at Westmesd - Gene Therapy Research Unit
A joint initiative of The New Children's Hospital and the Children's Medical Research Institute is
undertaking gene therapy clinical trials in Australia. Research projects include cancer
immunotherapy, vector development, HIV infection, targeting gene delivery to specific tissue types.
http://www.chw.edu.au/prof/services/genetherapy/
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Inherinet
Consortium of researchers from European institutions focused on developing gene therapy of
hematopoietic stem cells for inherited diseases.
http://www.inherinet.org
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European Society for Gene Therapy - ESGT
Organises annual meetings in Europe to promote gene therapy research.
http://www.esgt.org
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Vectron Therapeutics AG
Ddevelops targeted drug and gene delivery technologies using advanced colloidal lipid technologies
to create invasomes and artificial virus-like envelopes.
http://www.vectrontherapeutics.com
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Molecular Virology Lab at Leiden University
The group performs basic research on viruses in order to facilitate the development of efficient
gene-transfer vectors for use in gene therapy. Current research concerns the human adenoviruses and
the systems required for producing recombinant-adenovirus vectors.
http://members.lycos.nl/molvir/
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Qugen Therapeutics Pte Ltd
Focuses on the development of novel gene-based technologies and products providing first proof of
principle data in pre-clinical models and then moving these technologies into the clinical setting.
http://www.qugen.com
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Selective Genetics
Aims to commercialize site-specific gene therapeutics and DNA devices for tissue repair and
regeneration.
http://www.selectivegenetics.com
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Gene Therapy Advisory Committee - GTAC
Advises on the ethical acceptability of proposals for gene therapy research on humans taking
account of the scientific merits and the potential benefits and risks, and provides advice to UK
Health Ministers on developments in gene therapy research.
http://www.doh.gov.uk/genetics/gtac
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Harvard Gene Therapy Initiative
Provides plasmid construction and virus production services for gene therapy in both research and
therapeutic applications.
http://hgti.med.harvard.edu
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Ark Therapeutics
A gene medicine company developing a broad range of products based on vascular and endothelial
molecular biology to treat vascular and circulatory diseases and cancer.
http://www.arktherapeutics.com/
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Centre for Gene Therapeutics - McMaster University
The mission of the Centre is to investigate, create and implement approaches utilizing the delivery
of genes as therapeutic agents in the treatment of human and animal disease.
http://www.fhs.mcmaster.ca/cgt
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Crucell
Formed through the merger of IntroGene B.V. and U-BiSys B.V. Crucell combines IntroGene's human
cell line platform, PER.C6, with U-BiSys' subtractive phage antibody-display selection technology,
called MAb'stract, providing a powerful and effective means to discover, develop and produce a
variety of biopharmaceuticals for the treatment of human diseases.
http://www.crucell.com/
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GenVec
An emerging biopharmaceutical company developing novel gene therapies for diseases where local
delivery of a therapeutic gene has potential benefits over currently available treatments. New
product development initiatives have been formalized for coronary artery disease with BioBypass
angiogen, restenosis prevention and cancer.
http://www.genvec.com
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BioVex
Gene delivery technology allowing pharmaceutical and genomics companies to assess the therapeutic
value of identified genes and also allows genes to be used as therapeutic products to treat
diseases from cancer to nervous and immune system disorders
http://www.biovex.com/
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Oxagen Limited
Perform genetic analysis on collections of family samples so as to understand the mechanisms and
causes of common diseases, in order to identify new drug targets, the validation of targets and the
development of diagnostics.
http://www.oxagen.co.uk/
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Japan Society of Gene Therapy
Facilitates interdisciplinary gene therapy research between investigators performing basic and
applied research and clinical trials through academic conferences, international symposia and
publications.
http://bizweb.justnet.ne.jp/~jsgt/
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Pittsburgh Human Gene Therapy Center
Fosters the growth and development of multidisciplinary preclinical and clinical research programs
aimed at the use of gene transfer technologies for the treatment of human disease.
http://www.pitt.edu/~rsup/phgt/
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Amsterdam Molecular Therapeutics
Focused on developing gene therapies for lipoprotein lipase disorder, inflammatory bowel disease
and neuroregeneration. Also provides contract development and manufacturing of gene and
cell-based products in their GMP facility.
http://www.amtbv.com
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Oxford Biomedica
Focuses on developing gene transfer technologies for human gene therapy, gene-based immunotherapy,
and gene-based drug discovery for cancer, AIDS, neurodegenerative and cardiovascular disease.
http://www.oxfordbiomedica.co.uk
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Valentis, Inc.
Developing gene-based therapeutics or "gene medicines" for the treatment of certain
cancers, neuromuscular, cardiovascular, rheumatological and pulmonary diseases.
http://www.valentis.com
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The Gene Delivery Group at the University of Birmingham
Works primarily on developing non-viral and polymer vectors for cancer gene therapy.
http://web.bham.ac.uk/can4psd4/
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University of North Carolina Chapel Hill Gene Therapy
Working to translate gene therapy research from the laboratory bench into Phase I clinical trials
for the treatment of hematologic and endocrine diseases. The center has a GMP facility for clinical
grade gene vector production.
http://www.med.unc.edu/genether/
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Alberta Gene Therapy Group
A network of researchers looking to foster collaboration in several therapeutic fields.
http://www.ualberta.ca/~britchie/agtg.html
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Copernicus Therapeutics, Inc.
Developing human gene therapy products for cystic fibrosis and hemophilia B and DNA vaccinations.
The company creates proprietary PLASmin Complexes which are efficient non-viral vectors and
REPLIsome vectors which allow replication of non-viral vectors.
http://www.cgsys.com/
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Genteric, Inc.
A rapidly growing biopharmaceutical company that develops novel gene-based methods to treat human
diseases.
http://www.genteric.com
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University of Minnesota Gene Therapy Center
Clinical gene therapy research programs focusing on metabolic disorders.
http://www.peds.umn.edu/Centers/gene/
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Human Gene Therapy
A reference resource for the latest information concerning human gene therapy.
http://www.humangenetherapy.com/
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Oxford University GeneMedicine Research Group
Focused on the development gene therapies for diseases of the airway, such as asthma, lung cancer
and in particular, cystic fibrosis (CF). Work includes formulation development, reagent production,
pre-clinical testing, and human clinical trials.
http://users.ox.ac.uk/~genemed/default.htm
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Transgene S.A.
Gene therapy company based in France is developing products for cancer and cystic fibrosis. Vector
platforms include adenovirus, retrovirus, vaccinia virus, cellular and synthetic vectors.
http://www.transgene.fr
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Salus Therapeutics, Inc.
Developed a series of proprietary technologies for the discovery, development and delivery of
nucleic acid-based therapeutics, including antisense and gene therapy drugs.
http://www.salusinc.com/
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Amaxa Biosystems
NucleofectorTM technology allows non-viral gene transfer directly into the nucleus of primary cells
and cell lines, thereby providing rapid target gene validation, acceleration of drug development
and new applications for gene therapy.
http://www.amaxa.com/
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Finnish Gene Therapy Society
Fosters the exchange of ideas between research groups and individual scientists, organizes
scientific meetings and training courses and informs general public about basic aspects and recent
developments in gene therapy.
http://www.uku.fi/english/organizations/fgts/index.htm
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Ceregene, Inc.
Pioneering genetic therapies for neurologic disorders.
http://www.ceregene.com
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VirRx, Inc.
A biotechnology company with a primary interest in cancer gene therapy.
http://www.virrx.com
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Center for Gene Therapy at the University of Michigan Medical Center
The center links basic science, clinical investigation and technology transfer endeavors in the
area of gene therapy, while also serving as a resource for information and education.
http://www.med.umich.edu/cgt/
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Virapur
Sells virus purification kits for gene therapy and virus research which do not require cesium
gradients.
http://www.virapur.com
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Cell Genesys
Focused on the development and commercialization of cancer vaccines and gene therapies to treat
various cancers including leukemia, hemophilia A and B, restenosis after angioplasty and
Parkinson's disease.
http://www.cellgenesys.com
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Puresyn, Inc.
Develops separation and purification processes for manufacturing gene delivery products such as
supercoiled plasmids, synthetic oligonucleotides, recombinant adenovirus and adeno-associated
virus.
http://puresyn.com
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REPLICor, Inc.
Development of technologies controlling DNA replication for applications in a platform for gene
therapy vectors and in the discovery of new therapeutic agents for cancer and tissue regeneration.
http://www.t2c2capital.com/replicor.htm
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Gene-Cell, Inc.
Focused on the targeted genetic modification of human somatic stem cells for gene therapy.
http://www.gene-cell.com
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Laboratory for Cancer Gene Therapy at Hamburg University
Gene therapy research program in colon, lung and bladder cancer are being conducted by the
laboratory of Dr. Andreas Block.
http://www.uke.uni-hamburg.de/Clinics/InternalMed/Kernklinik/Pages/block/bl_introduction.htm
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Collateral Therapeutics
A product-driven cardiovascular gene therapy company, focused on the discovery, development and
commercialization of novel and innovative non-surgical gene therapy products.
http://www.collateralthx.com
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AcuVector Group Inc.
Developed advanced technologies to formulate and test gene-therapy vaccines and is in the process
of developing commercial product lines
http://www.ispex.ca/acuvector
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Retroscreen Ltd.
Virological research and clinical trials company that work on most model systems and carry out
projects from laboratory based assays to full ethics committee approved clinical trials.
http://www.retroscreen.com
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American Society of Gene Therapy
Organization dedicated to promoting gene therapy research through public education, scientific
meetings and scientific committees targeting specific therapeutic areas and also vector technology.
http://www.asgt.org
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Office of Biotechnology Activities at the NIH
Monitors scientific progress and ethical issues in basic and clinical research involving
recombinant DNA and human gene transfer at NIH funded institutions.
http://www.nih.gov/od/oba/
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Gene Therapy at MD Anderson Cancer Center
The human cancer gene prevention and therapy program conducts basic research, clinical trials and
vector development.
http://www.mdanderson.org/depts/genetherapy/
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Molecular Medicine Bioservices, Inc.
A development and manufacturing company based in San Diego dedicated to the production of
high-quality, clinical grade, gene and cell based therapeutics and reagents.
http://www.molecularmed.com/
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UCSD Program in Human Gene Therapy
Facilitates the application of basic and pre-clinical research to the treatment of human disease
through vector development and gene discovery and characterization.
http://medicine.ucsd.edu/gt/index.html
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Introgen Therapeutics, Inc.
A leader in the development of gene therapy products to treat a variety of cancers.
http://www.introgen.com
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Center for Cell and Gene Therapy
Clinical and fundamental research, job opportunities, faculties' interests and links to gene
therapy sites - joint project of Baylor College of Medicine, Texas Children's Hospital, and The
Methodist Hospital in Houston, TX.
http://www.bcm.tmc.edu/genetherapy
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InvivoGen
Tools for gene therapy and cancer research including plasmid and cloning vectors, gene collections,
selection antibiotics, mycoplasma curing kit, and prodrugs.
http://www.invivogen.com
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The Journal of Gene Medicine
An information source on all aspects of human gene therapy. It offers an international
comprehensive summary of both published and ongoing clinical trials. Also includes reviews on basic
and therapeutic issues.
http://www.wiley.co.uk/genetherapy/
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Gene Therapy Systems
Commercial firm providing reagents for gene delivery and gene therapy research, including unique
pGeneGrip fluorescently labeled plasmid vectors and GenePORTER cationic lipid transfection reagent.
http://www.genetherapysystems.com
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GeneBrowser
Commercial site focusing on the current state of retroviral vector development for gene therapy
with numerous helpful links - from Nature Technology Corp.
http://www.natx.com
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