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Companies - A

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 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).
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.

Companies - B

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.

Companies - C

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.
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.
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 Biologics Ltd
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.

Companies - F

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.

Companies - G

Gene Medicine Japan Inc.
Japanese based company committed to research and develop innovative gene medicine technologies such as adenoviral vectors and cell processing.
Genlantis
Commercial firm focused on developing reagents for transfection, gene expression, gene Silencing, RNA interference, primary cell culture, and sample preparation in proteomics research.
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.

Companies - I

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.
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. .

Companies - L

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.

Companies - M

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.

Companies - N

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.
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.

Companies - O

Ocata Therapeutics, Inc
Clinical stage biotechnology company focused on the development and commercialization of regenerative medicine and cell therapy technologies.
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 Genetics Ltd
Oxford Genetics provides a wide range of genetic materials for the creation of plasmid and virus based gene expression systems. Using a modular DNA assembly system, creating complex genetic systems is made significantly more efficient.
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.

Companies - P

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.

Companies - R

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.

Companies - S

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.
SiBiono GeneTech Co., Ltd.
Chinese biotechnology company commercialized the first gene therapy product Gendicine, a recombinant Human Ad-p53, used for cancer treatment.
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.
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.

Companies - T

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.

Companies - U

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.

Companies - V

  VBL Therapeutics
VBL Therapeutics is a publicly traded (NASDAQ: VBLT), late-stage clinical biotechnology company focused on the discovery, development and commercialization of first in class treatments for cancer. VBL’s clinical pipeline is based on two distinct, proprietary platform technologies that leverage the body’s natural physiologic and genetic regulatory elements. To date, VBL has developed two programs based on these platforms – an oncology program and an anti-inflammatory program. VBL is based in Tel Aviv, Israel.
  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.
  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.
    

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.


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