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Addgene
Non-profit organization working with hundreds of laboratories to assemble a high-quality library of published plasmids for use in research and discovery. By linking plasmids with articles, scientists can always find data related to the materials they request.
Advanced Cell & Gene Therapy, LLC
Consulting firm in cell therapy, gene therapy and tissue therapies, serving biotechnology companies, clinical laboratories, and biotechnology investors.
Advanced Cell Technology
Biotechnology company focused on applying stem cell technology in the field of regenerative medicine to bring effective, patient-specific therapies to the bedside.
Advanced Vision Therapies, Inc.
Gene transfer technology for the treatment of eye diseases like Wet Age-Related Macular Degeneration (AMD), Diabetic Proliferative Retinopathy (DPR), Retinitis Pigmentosa (RP) and Dry Age-Related Macular Degeneration.
AlphaVax Human Vaccines, Inc.
AlphaVax is developing a new vaccine technology, alphavaccines, with broad applications against cancer and infectious disease, like pandemic influenza and cytomegalovirus.
Altogen Biosystems
Life sciences company dedicated to the development, marketing and manufacture of cell line specific transfection reagents for intracellular delivery of biomolecules (siRNA and plasmid DNA).
Amaxa AG
Innovative tools for non-viral delivery of genes and the transfer of other charged substrates into cells.
Amsterdam Molecular Therapeutics (AMT)
Gene therapy company developing therapies of severe (orphan) diseases like metabolic disorders, LPL deficiency using AAV vectors, and disorders of the central and peripheral nervous systems.
Applied Tissue Technologies LLC
Technologies focus on direct methods of treating infection in skin wounds, transplanting skin to wounds, and delivering genes to skin cells.
Ark Therapeutics Ltd.
Gene therapy company focuses on areas of high unmet clinical need in vascular disease, wound care and cancer. Products in late stage clinical development: Cerepro®, Vitor™ and Trinam®
Asklepios BioPharmaceutical Inc.
Biotechnology company engaged in the development and delivery of novel protein and cellular based therapies through design of Biological Nano Particles (BNP). BNPs are synthetically designed from components of different Adeno Associated Viruses as well as other parvoviruses.
Athersys, Inc.
Clinical stage biopharmaceutical company with lead programs in the areas of obesity, cardiovascular disease (myocardial infarction), and bone marrow transplant support.
Austrianova
Focused on the development and manufacture of highly innovative tumour treatments based on localized cell therapies. Reference product NovaCaps® is the first encapsulated cell product targeting solid tumours.
Avaris AB
Swedish biotechnology company that develops and commercializes gene and cell therapy products in the field of chronic infections, cancer and inherited diseases.
 
Bavarian Nordic A/S
Biopharmaceutical company developing and producing innovative vaccines by using vaccine (MVA) vectors for the development of vaccines against various infectious diseases such as smallpox, HIV/AIDS, as well as against breast and prostate cancer.
BioCardia Inc
BioCardia's Helix Biotherapeutic Delivery business remains committed to being the platform of choice for delivery of therapeutic agents to the myocardium.
Bioheart, Inc.
Biotechnology company focused on the discovery, development and, subject to regulatory approval, commercialization of autologous cell therapies for the treatment of chronic and acute heart damage. Lead product candidate is MyoCell.
BioProtein Technologies
Contract Manufacturing Organisation specializing in the production of human therapeutic proteins & vaccines in the milk of transgenic rabbits.
BioVex Limited
Biotechnology company developing clinical stage treatments for cancer and the prevention of infectious disease. Phase III clinical trial evaluating its lead product, OncoVEXGM-CSF in previously treated patients with metastatic melanoma.
 
Cardium Therapeutics
Medical technology company primarily focused on the development and commercialization of novel biologic therapeutics and medical devices for cardiovascular and ischemic disease.
Cellectis SA
Cellectis proprietary technologies and platform are based upon a naturally occurring class of DNA endonucleases, the Meganucleases and DNA recombination.
Cellerant Therapeutics Inc.
Biotechnology company with a portfolio of products based on the regulation of the hematopoietic (blood-forming) system. Cellerant is developing a novel, cell-based medicine (Myeloid Progenitors / CLT-008) as a treatment for chemotherapy- and radiation-induced neutropenia as well as for Acute Radiation Syndrome.
CellGenix Technologie Transfer GmbH
CellGenix develops, manufactures and markets cell and protein therapeutics for cancer and orthopedic patients, as well as high-quality reagents for therapeutic ex vivo cell processing.
Cellprep S.A.
Argentine biotechnology company committed to the research and development of cell processing techniques and practices applied in different fields of medicine.
Cenix BioScience GmbH
Cenix is a contract research provider specialized in RNAi- and miRNA-based Target Discovery, Target Validation and Drug Mode of Action studies combined with high-content phenotypic analyses.
Ceregene Inc.
Biotechnology company focused on developing neurotrophic growth factor treatments for major neurodegenerative disorders, like Alzheimer’s disease and Parkinson’s disease, using a proprietary AAV-based gene delivery technology.
Clontech
Biotechnology supply company offering adenoviral-mediated gene transfer products, like Adeno-X Tet-On®, Adeno-X Tet-Off® Expression Systems for inducible adenoviral protein expression, Adeno-X Virus Purification Kit, Adeno-X Rapid Titer Kit, and Adeno-X Marker Viruses.
Cobra Biomanufacturing
Contract manufacturing organisation focusing on the production of Protein, Viral and DNA-based therapeutics at cGMP grade.
Copernicus Therapeutics Inc.
Biopharmaceutical company utilizing its breakthrough DNA nanoparticle technologies to produce safe and effective DNA drugs. Copernicus' lead products is for cystic fibrosis and hemophilia B.
Cyto Pulse Sciences, Inc.
Medical device and treatment development company dedicated to advancing quality of life through novel electric field based technologies for cancer treatment, gene therapy and infectious disease applications.
CytoGenix Inc.
Biopharmaceutical company that develops and markets innovative compounds and services based on its proprietary gene regulating and DNA production technologies. CytoGenix has products in development including an antimicrobial compound, DNA vaccines and anti-herpes and anti-inflammatory topical creams.
 
Enzo Biochem, Inc.
Biotechnology company focused on the development of enabling technologies in the areas of gene regulation and gene modification.
Epeius Biotechnologies Corporation
Epeius Biotechnologies have developed a targeted delivery system (TDS) that can transport genes or other therapeutic agents directly to diseased areas in the body. This technology has enabled the company to develop Rexin-G™, the first tumor-targeted, injectable gene delivery system.
Expression Genetics Inc.
Biopharmaceutical company developing polymeric delivery systems to be used to create products for treatment of human diseases. Our delivery vehicles, based on a synthetic biocompatible polymeric system can be used to deliver therapeutic genes, inhibitory RNA (siRNA & shRNA) and small molecules.
 
Florida Biologix
A CMO that provides process development solutions, customized manufacturing, aseptic filling and other cGMP services for Phase I/II mammalian cell derived products such as vaccines, gene therapeutics, and protein products.
 
Gene Medicine Japan Inc.
Japanese based company committed to research and develop innovative gene medicine technologies such as adenoviral vectors and cell processing.
Genetix Pharmaceuticals Inc.
Biotechnology company developing effective gene therapies for the treatment of serious, chronic human diseases, like hereditary blood disorders.
Genlantis
Commercial firm focused on developing reagents for transfection, gene expression, gene Silencing, RNA interference, primary cell culture, and sample preparation in proteomics research.
GenoSafe
CSO specialized in the evaluation of the quality, efficacy and safety of gene therapy products. A real partnership from research stages to clinical phases. Study design, development/validation of analytical methods, and product testing: - Control of viral vectors batches (rAAV, rHIV, rMLV), - Preclinical evaluation - Clinical trial: patients follow-up.
GenVec, Inc.
Biopharmaceutical company developing gene-based therapeutic drugs and vaccines. Lead product TNFerade™ is used in locally advanced pancreatic cancer, rectal cancer, melanoma, and head and neck cancer. GenVec uses adenovector technology to develop vaccines for infectious diseases.
 
Ichor Medical Systems, Inc.
Ichor is dedicated to the clinical application and commercialization of electroporation technology (TriGrid™ Delivery System) for the delivery of DNA drugs and vaccines to treat and prevent debilitating or life threatening diseases.
Intercytex Limited
Healthcare company using its proprietary cell therapy technology to develop innovative regenerative medicine products to restore skin and hair.
Introgen Therapeutics, Inc.
Biopharmaceutical company focused on the development of targeted molecular therapies for the treatment of cancer and other diseases using tumor suppressors, cytokines and other targeted molecular agents like ADVEXIN® and INGN 241.
Invitrogen Corporation
Biotechnology supply company offering ViraPower™ Lentiviral and Adenoviral Expression Systems that can be used for highly-efficient delivery of RNAi or expression constructs in mammalian cell lines.
InvivoGen
Biotechnology company focused on providing innovative products to the scientific community worldwide. Primary objective is to take advantage of cutting-edge technologies to provide research tools that are high quality and easy to use. .
Ixion Biotechnology
Biotechnology company that is developing therapies focused on treatment of metabolic disorders, like primary hyperoxaluria, recurrent kidney stones, and diabetes.
 
Lentigen Corporation
Biotechnology supply company focused on custom manufacture of lentiviral vectors by using LentiMax Production System.
Lysogene
Lysogene is a clinical stage pioneering biotechnology company in the field of gene therapy targeting severe genetic pathologies of the central nervous system in children.
 
MaxCyte, Inc.
Cell therapeutics company developing cell-based therapies. The company's proprietary, non-viral, ex vivo cell loading technology is used in partnered therapeutic programs in oncology, pulmonary, metabolic and infectious diseases.
MediGene, Inc.
MediGene concentrates on commercializing novel drugs in three therapeutic areas: cancer, autoimmune diseases, and skin diseases.
Microbix
Biopharmaceutical company specialized in developing biological technologies in the field of biotherapeutics, vaccines and non-therapeutic biologics, including Adeno-associated virus vectors (rAAV) and Adenoviral vectors.
Mirus Corporation
Biopharmaceutical company focused on developing innovative nucleic acid based technologies and products. Under development are RNAi gene silencing therapies based on Mirus’ proprietary Dynamic PolyConjugates™ delivery platform.
Molecular Medicine, LLC
Company is dedicated to providing a variety of development, manufacturing, and ancillary services for the viral vaccine and viral therapeutic marketplace.
MolMed S.p.A
Medical biotechnology company focused on discovery, R&D, and clinical validation of innovative therapies for the treatment of cancer.
Mologen Holding AG
Biotechnology company researching and developing innovative DNA-based drugs, inclding improving existing immunological drugs and therapies to combat cancer and developing state-of-the-art vaccines for the prevention and treatment of serious infectious diseases.
 
NanoCor Therapeutics, Inc.
Biotechnology company founded to create viable intracellular genetic protein therapy for the treatment of Chronic Heart Failure. NanoCor has obtained rights to the Biological NanoParticle (BNP®) technology platform. BNPs are derived from Adeno-Associated Viral vectors.
Nature Technology Corporation
Bioengineering technology company, providing industry solutions for design, research, process development and manufacturing of biologics, including gene-therapeutics and vaccines.
Neurologix Inc.
Biotechnology company developing gene transfer therapies for Parkinson's disease, epilepsy and Huntington's by using the adeno-associated virus ("AAV") vector.
Neurotech SA
Biotechnology company dedicated to the development of sight-saving therapeutics for chronic retinal diseases. Neurotech’s lead product (NT-501) is in clinical development for the treatment of retinal degeneration and geographic atrophy.
Novocell Inc.
Stem cell engineering company dedicated to creating, delivering, and commercializing cell and drug therapies to treat chronic diseases including diabetes.
 
Omnia Biologics, Inc.
Omnia Biologics provides customized manufacturing, cGMP services, and process development solutions for the development of vaccines, gene therapeutics, and biodefense products.
Onyx Pharmaceuticals, Inc.
Biotechnology company developing small molecule cancer treatments. Their lead product is Nexavar® (sorafenib) tablets, a novel, oral multiple kinase inhibitor that targets tumor cell proliferation and angiogenesis.
ORCA Therapeutics B.V.
Netherlands biopharmaceutical company focusing on the development of new anti-cancer treatments using Oncolytic Replication Competent Agents (ORCA).
OrphageniX
OrphageniX is dedicated to treating Orphan Diseases utilizing Targeted Gene Alteration (TGA). The Company will focus its efforts on developing oligotherapy technologies.
Oxford BioMedica
Biopharmaceutical company specialising in the development and commercialisation of innovative gene-based medicines for treatment for cancer (TroVax®), Parkinson’s disease and retinopathy.
 
Polygenetics Inc.
Pharmaceutical company with emphasis on specialty generics. The company develops and offers combination products comprised of active pharmaceutical ingredients loaded into highly porous polymers.
PrimeGen Biotech LLC.
Biotechnology company dedicated to the research and therapeutic applications of adult stem cells, leading to individualized patient therapies. The company is specialized in two core technologies: germ line stem cells and therapeutic reprogramming.
Progenitor Cell Therapy, LLC.
Contract Manufacturing company that is dedicated to bridging the gap between the concepts in the cell therapy industry and their therapeutic application in patient care by providing quality product service solutions.
Proneuron Biotechnologies, Inc.
Biopharmaceutical company developing therapies for neurological disorders, including spinal cord injuries (SCI), brain trauma, and neurodegenerative diseases such as ALS, Huntington’s and Alzheimer’s diseases.
Puresyn, Inc.
Develops separation and purification processes for manufacturing gene delivery products such as supercoiled plasmids, synthetic oligonucleotides, recombinant adenovirus and adeno-associated virus.
 
Regulon Inc.
Biotechnology company committed to the development of anti-cancer pharmaceuticals. Their objective in developing a broad range of low-toxicity drugs to be used in cancer therapy is based on liposomal encapsulation technology.
Reneuron
ReNeuron is a pioneer in stem cell research and development focused on cell therapy treatments designed to reverse the effects of major diseases such as stroke, diabetes and diseases of the retina.
REPLICor Inc.
Biopharmaceutical company developing entry inhibitors for the treatment of hepatitis C (HCV) and prevention of HCV re-infection following liver transplantation. The company also targets the hepatitis B and influenza markets.
 
Sernova Corp.
Cellular therapy company with platform technology for creating a local immunoprivileged site enabling the therapeutic transplantation of cells and cell-based gene therapy.
Shanghai GenePharma
The company has internationally lead siRNA chemical synthesis complete core technology, including RNA monomer synthesis technology, common and chemically modified siRNA oligo synthesis technology.
Shanghai Telebio Biomedical Co., Ltd.
Shanghai based biomedical company involved in gene therapy. Main product is the Televector Lentiviral vector easy-high production kit. Information only in Chinese.
SiBiono GeneTech Co., Ltd.
Chinese biotechnology company commercialized the first gene therapy product Gendicine, a recombinant Human Ad-p53, used for cancer treatment.
Sirna Therapeutics
Sirna Therapeutics is at the forefront of the effort to create RNAi- based therapies and leverage the vast potential of this technology to ultimately treat patients.
Stem Cell Sciences
Research and development company specialising in the provision of stem cells and stem cell technologies for research and the clinic.
StemCells Incorporated
StemCells, Inc. is focused on the discovery and development of stem cell therapeutics to treat damage to or degeneration of major organ systems such as the Central Nervous System, Liver and Pancreas.
Stratagene
Biotech company supplying reagents for molecular biology applications. Stratagene has expanded into the viral gene transfer market, offering systems based on AAV, Adenovirus (AdEasy system) and Retrovirus.
Sunway Biotech Co., Ltd.
This Shanghai based biotechnology company is the main developer of the H100 technology and genetically engineered adenoviruses for cancer treatments. It has successfully developed drugs to treat cancer such as H101, H102, and H103.
Supratek Pharma Inc.
Pharmaceutical company dedicated to the commercialization of anticancer therapeutics desperately needed by cancer patients with tumors that are resistant to available therapies.
 
Targeted Genetics Corporation
Biotechnology company developing molecular therapeutics for the treatment for inflammatory arthritis, HIV/AIDS, congestive heart failure and Huntington's disease.
TheraCyte, Inc.
Company developing cell-based therapeutic products for treating chronic and recurrent diseases, including diabetes, infection, protein deficiencies and immunological disorders.
TheraVitae Co., Ltd.
Biotechnology company at the forefront of regenerative medicine with a mission to make the benefits of adult stem cell therapy accessible to people suffering from diseases like heart disease and peripheral artery disease.
Transgene S.A.
Biopharmaceutical company dedicated to the discovery and development of gene-based therapeutic vaccines and immunotherapy products for the treatment of cancer and infectious diseases.
 
uniQure
Company involved in the research and early development of human gene based therapies. uniQure has a product pipeline of several gene therapies in development: hemophilia B, acute intermittent porphyria, Parkinson’s disease and Sanfilippo. uniQure is the world leader for manufacturing of AAV-based products on a commercial scale.
 
Vector Gene Technology Company, Ltd.
VGTC is a leader in recombinant adeno-associated virus (rAAV) large-scale manufacturing. Its novel AAV package and purification systems make the producing of AAV to be one of the most cost-effective AAV producing systems.
Vectorite Biomedica Inc.
Biotechnology company focusing on the development of therapeutic lentivirus vector technology for the delivery of short interfering RNA molecules.
Vecura
Contract manufacturer of gene therapy vectors and cell therapy products for clinical trials. The laboratory is a part of the Gene Therapy Center at Karolinska University Hospital.
Vical Incorporated
Biotechnology company developing biopharmaceutical products based on DNA delivery technologies for the prevention and treatment of serious or life-threatening diseases, like infections diseases and cancer.
VirRx, Inc.
Biotechnology company with a primary interest in cancer gene therapy. Its broad mission is to develop genetically modified "smart bomb" adenoviruses for use in treating human cancers.
VIRxSYS Corporation
Biotech company whose mission is to develop gene therapies using its proprietary lentiviral vector delivery system. The company is developing the first application of its gene therapy technology against HIV, VRX496.
 
Information Companies

Overview of biotechnology companies involved in gene therapy and/or cell therapy, as well as contract research organizations (CRO) and medical supply companies.

The provided information about the companies is just a summary of the information giving on their websites and is therefore not always complete, correct and/or exhaustive. Please visit the companies websites for a complete picture of the company.

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A seperate section lists gene therapy and biotechnology companies in China.

Like to work for a company? Find here the perfect job in gene therapy.


The Vector Directory

The Vector Directory details UK, EU and US suppliers of the vectors commonly used in gene therapy, and gives guidance to researchers seeking to have vectors manufactured for clinical trials. It is intended to help researchers bring the advances in genetics more quickly to the patient's bedside.

The Vector Directory is commissioned by the UK Department of Health. Published: 30 October 2007.