Silver

AFM Telethon

AFM (French Muscular Dystrophy Association) has a single objective: to defeat neuromuscular diseases, which are devastating muscle-wasting diseases. Created in 1958 by a group of patients and their families, and recognised as being of public utility in 1976, it has set itself two missions: curing neuromuscular diseases and reducing the disabilities they cause.

www.afm-france.org

e-Zyvec

e-Zyvec is a startup offering services in molecular biology to research laboratories, CROs and Biotech industries that are using genetic engineering approaches.
Our offer is to produce tailor-made ready-to-use gene expression vectors (plasmids) and the expertise to design such molecules so they will really fit our customers’ needs. We are exclusively exploiting a patented DNA assembly method that enable us to modularly assemble vectors from up to 12 DNA fragments in an ordered and seamless way in a single-tube single-step reaction. Our process has been optimized and streamlined so we can carry out multiple assemblies simultaneously.
Building vectors with us is unrestrictive, easy, fast and accurate.
 

Genethon

Genethon, is a pioneer and a leader in the field of gene therapy for rare genetic diseases. Genethon has multiple ongoing programs at clinical, preclinical and research stage for neuromuscular, blood, immune system, and liver diseases. Several of these programs are pursued by Genethon as sponsor, others have been licensed to leading biotech and pharma players in the gene therapy arena such as Avexis/Novartis (to which Genethon licensed the founding patents for Zolgensma), Audentes, Gensight Biologics, Orchard Therapeutics, Spark.
Genethon has around 200 scientists and experts in its R&D center in Evry (France), covering R&D, bioprocess development, clinical and regulatory.
Genethon is a non-profit organization created in 1990 by the AFM-Telethon (French Muscular Dystrophy Association) which is its main funding source.
 

Miltenyi Biotec

Miltenyi Biotec is a global provider of products and services that advance biomedical research and
cellular therapy. Since 1989, we have developed innovative and reliable technologies for scientists
and clinicians around the world. Our integrated portfolio of tools covers techniques of sample
preparation, cell separation, flow cytometry, cell culture, molecular analysis, and preclinical imaging.
The MACS® brand has set standards worldwide and is trusted across basic, translational, and clinical
research settings. Our expertise covers research areas like immunology, stem cell biology,
neuroscience, and cancer, and clinical research areas that include hematology, graft engineering, as
well as apheresis. We are committed to supporting scientists and the scientific community – from
comprehensive technical support to training at our MACS Academy. Today, we are more than 2,000
employees in 22 countries – all dedicated to providing solutions that empower scientific discovery
and advance cellular therapy.
 

Bronze

bluebird bio

Bluebird bio is developing innovative gene therapies for severe genetic disorders. At the heart of bluebird bio’s product creation efforts is its broadly applicable gene therapy platform for the development of novel treatments for diseases with few or no clinical options. The company’s novel approach uses stem cells harvested from the patient’s bone marrow into which a healthy version of the disease causing gene is inserted.

Gilead

Gilead Sciences, Inc. is a research-based biopharmaceutical company that discovers, develops and commercializes innovative medicines in areas of unmet medical need.  The company strives to transform and simplify care for people with life-threatening illnesses around the world. Gilead has operations in more than 35 countries worldwide, with headquarters in Foster City, California, USA.
Kite, a Gilead Company, is a biopharmaceutical company based in Santa Monica, California, USA. Kite is engaged in the development of innovative cancer immunotherapies. The company is focused on chimeric antigen receptor and T cell receptor engineered cell therapies.
 

Lysogene

Lysogene is a global biotechnology company, a leader in the basic research and clinical development of gene therapy for neurodegenerative disorders. Its mission is to radically improve the health of patients suffering from incurable life threatening conditions by developing AAV vectors that have demonstrated their effectiveness in safely delivering genetic material to the central nervous system.

Lysogene’s most advanced product candidate is rAAV vector serotype rh.10 carrying the human N-sulfoglucosamine sulfohydrolase (hSGSH) for the treatment of MPS IIIA.The recently completed Phase I/II study in four MPS IIIA children demonstrated that the gene therapy and neurosurgical procedure is safe, well tolerated and exploratory efficacy profiles are encouraging (Tardieu 2014).  A phase III clinical study is under preparation.

Following a successful Series A financing round, Lysogene is expanding its pipeline to GM1-gangliosidosis and other genetic diseases affecting the central nervous system.

Plasmid Factory

PlasmidFactory GmbH & Co. KG is a globally active biopharmaceutical company, founded in Bielefeld, Germany, in 2000. As a leading contract manufacturing organization (CMO) for plasmid and minicircle DNA, it has a strong customer base in the fields of cancer research, gene and cell therapy, CAR-T-cell development, and vaccination. PlasmidFactory produces plasmids and minicircles according to customer's requirements in modern laboratories with high quality standards. In addition to its In Stock products, e.g. for the worldwide exclusively offered pDG/pDP AAV 2-plasmid-system, and custom manufacturing of plasmid and minicircle DNA, PlasmidFactory focuses its R&D efforts on its core competencies in the production, analysis, application, and storage of DNA.

SCIEX

SCIEX helps to improve the world we live in by enabling scientists and laboratory analysts to find answers to the complex analytical challenges they face. The company's global leadership and world-class service and support in the capillary electrophoresis and liquid chromatography-mass spectrometry industry have made it a trusted partner to thousands of the scientists and lab analysts worldwide who are focused on basic research, drug discovery and development, food and environmental testing, forensics and clinical research.
 
With over 40 years of proven innovation, SCIEX excels by listening to and understanding the ever-evolving needs of its customers to develop reliable, sensitive and intuitive solutions that continue to redefine what is achievable in routine and complex analysis. 
 

VIVET Therapeutics

Vivet Therapeutics is a biotechnology company developing novel gene therapy treatments for rare, inherited metabolic diseases.
 
Vivet’s lead program, VTX-801, is a novel investigational gene therapy for Wilson disease which has been granted Orphan Drug Designation (ODD) by the Food and Drug Administration (FDA) and the European Commission (EC). This rare genetic disorder is caused by mutations in the gene encoding the ATP7B protein, which reduces the ability of the liver and other tissues to regulate copper levels causing severe hepatic damage, neurologic symptoms and potentially death.
 
Vivet is building a diversified gene therapy pipeline based on novel adeno-associated virus (AAV) with 5 preclinical programs and new technologies addressing key challenges of AAV-based gene therapy related to the sustainability of expression in young patients and immunomodulation for potential retreatment. Vivet develops these technologies through its partnership with, and exclusive licenses from, the Fundación para la Investigación Médica Aplicada (FIMA), a not-for-profit foundation at the Centro de Investigación Medica Aplicada (CIMA), University of Navarra based in Pamplona, Spain.