List of Publicly Traded Gene Therapy Companies Listed on Major U.S. The company offers the production of plasmids and minicircles in several quality grades: Research Grade and ccc Grade for research purposes and pre-clinical applications, High Quality (HQ) Grade as starting material for e.g. The company expects its lead candidate for an HIV cure to enter the clinic in 2018, and it has developed a patented immuno-oncology approach to attack a variety of epithelial cancers that is expected to enter trials soon.Technologies focus on direct methods of treating infection in skin wounds, transplanting skin to wounds, and delivering genes to skin cells.Gene therapy company focuses on areas of high unmet clinical need in vascular disease, wound care and cancer. George Clinical provides the full range of clinical trial services to pharmaceutical, medical device and biotech customers, for all trial phases, registration and post-marketing trials. Their objective in developing a broad range of low-toxicity drugs to be used in cancer therapy is based on liposomal encapsulation technology.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.Biopharmaceutical company developing entry inhibitors for the treatment of hepatitis C (HCV) and prevention of HCV re-infection following liver transplantation.
VBL’s clinical pipeline is based on two distinct, proprietary platform technologies that leverage the body’s natural physiologic and genetic regulatory elements.
Products in late stage clinical development: Cerepro®, Vitor™ and Trinam®An Amsterdam based clinical stage, biopharmaceutical company, is developing local gene therapy for inflammatory diseases, using viral mediated gene transfer. Copernicus' lead products is for cystic fibrosis and hemophilia B.Biotechnology company focused on the development of enabling technologies in the areas of gene regulation and gene modification.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.Japanese based company committed to research and develop innovative gene medicine technologies such as adenoviral vectors and cell processing. Clinical stage biopharmaceutical company with lead programs in the areas of obesity, cardiovascular disease (myocardial infarction), and bone marrow transplant support.Focused on the development and manufacture of highly innovative tumour treatments based on localized cell therapies. 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.
Luxturna is an adeno-associated virus (AAV)-based gene therapy, and is considered as the first true gene therapy as it works by delivering a normal copy of the gene of interest directly to the host cell ( in this case retinal cells) using a vector (AAV vector serotype 2 (AAV2)).
CTRL + SPACE for auto-complete. The publicly traded companies on this list are engaged in gene therapy which uses genes in the attempt to treat or prevent a wide range of diseases. The high saturation of gene and cell therapy companies with therapeutics nearing clinical-stage could make Maryland the nation’s number one leader in medical innovation in the next decade. In cell therapy… For a better look into Maryland’s gene and cell therapy industry, here is a list of 170 companies … Their lead product is Nexavar® (sorafenib) tablets, a novel, oral multiple kinase inhibitor that targets tumor cell proliferation and angiogenesis.Netherlands biopharmaceutical company focusing on the development of new anti-cancer treatments using Oncolytic Replication Competent Agents (ORCA).OrphageniX is dedicated to treating Orphan Diseases utilizing Targeted Gene Alteration (TGA). We are the unique providers of a PEI suitable for use as raw material in GMP processes.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.Develops separation and purification processes for manufacturing gene delivery products such as supercoiled plasmids, synthetic oligonucleotides, recombinant adenovirus and adeno-associated virus.Biotechnology company committed to the development of anti-cancer pharmaceuticals. Two gene therapy candidates on ARM’s list are not included. uniQure is the world leader for manufacturing of AAV-based products on a commercial scale.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.
uniQure has a product pipeline of several gene therapies in development: hemophilia B, acute intermittent porphyria, Parkinson’s disease and Sanfilippo. The first target indication is in the field of rheumatic diseases. Exchanges.
You can also make a donation by PayPal and support the Gene Therapy Net website.Non-profit organization working with hundreds of laboratories to assemble a high-quality library of published plasmids for use in research and discovery. Now, more than 30 companies and 60 labs in the region are inventing ways to empower the body to combat disease through cell and gene therapy. One is CMB305, a prime-boost cancer vaccine designed to target tumors that express NY-ESO-1.