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Blue Bird 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.
Genethon develops and manufactures gene therapy products for rare diseases with the goal of making these innovative treatments available to patients. To meet this challenge, Genethon has assembled the technical and human resources needed to accelerate the medical application of scientific discoveries arising from fundamental research. Strong translational research programmes (at preclinical and clinical stages) engage multi-disciplinary teams and are supported by a first-rate technological platform and cGMP capacity. Genethon currently sponsors several early-phase gene therapy clinical trials (including an international trial). In 2011 Genethon will open Genethon BioProd, the world’s largest production plant for clinical-grade gene therapy products.
PERSIST project explores the use of highly innovative gene-modifying and delivery technologies, and capitalises on recent discoveries in gene expression control to develop radical solutions to the problem of precisely controlling the fate and expression of exogenous genetic information in gene therapy, with applications in these and other deadly diseases. The project combines more than 20 of Europe’s outstanding experts from eight countries in the field of genetic engineering for persisting gene expression.
Founded in France in 1999, the Cellectis Group is based on a highly specific DNA engineering technology. Its application sectors are human health, agriculture and bio-energies. Co-created by André Choulika, its Chief Executive Officer, Cellectis is today one of the world leading companies in the field of genome engineering. The Group has a workforce of 230 employees working on 5 sites worldwide: Paris & Evry in France, Gothenburg in Sweden, St Paul (Minnesota) & Cambridge (Massachusetts) in the United States. Cellectis achieved in 2011 €19M revenues and has signed more than 80 industrial agreements with pharmaceutical laboratories, agrochemical and biotechnology companies since its inception. AFM, Dupont, BASF, Bayer, Total, Limagrain, Novo Nordisk… are some of the Group’s clients and partners.
Since 2007, Cellectis has been listed on NYSE-Euronext Alternext market (code: ALCLS) in Paris.
Amgen discovers, develops, manufactures, and delivers innovative human therapeutics. A biotechnology pioneer since 1980, Amgen was one of the first companies to realize the new science’s promise by bringing safe, effective medicines from lab to manufacturing plant to patient. Amgen therapeutics have changed the practice of medicine, helping millions of people around the world in the fight against cancer, kidney disease, rheumatoid arthritis, bone disease, and other serious illnesses. With a deep and broad pipeline of potential new medicines, Amgen remains committed to advancing science to dramatically improve people’s lives.
BD Biosciences is a world leader in bringing innovative diagnostic and research tools to life science researchers, clinical researchers, laboratory professionals and clinicians who are involved in basic
research, drug discovery and development, biopharmaceutical production and disease management. The BD Biosciences segment is focused on continually advancing the science and applications associated with
cellular analysis and products that help grow living cells and tissue.
· Fluorescence-activated cell sorters and analyzers
· Monoclonal antibodies and kits for cell analysis
· Reagent systems for life science research
· Cell imaging systems
· Laboratory products for tissue culture and fluid handling
· Cell culture media and supplements for biopharmaceutical
Fanconi anemia (FA) is a rare inherited syndrome characterized by the early development of bone marrow failure and increasing predisposition to cancer with age. Allogeneic hematopoietic cell transplantation (alloHCT) is the only curative therapy for hematopoietic manifestations of FA, although associated with complications arising from myeloablation, graft versus host disease and increased incidence of squamous cell carcinoma.
The genetic correction of autologous hematopoietic stem cells (HSC) with lentiviral vectors constitutes a recent and safe alternative for the treatment of different genetic diseases affecting mature cells from different tissues and/or committed progenitors of the hematopoietic system. One of the key features of FA that make it a unique disease for gene therapy approaches rely on the characteristic proliferation defect that is already evident in the very primitive HSCs. Thus, a marked survival advantage would be expected from corrected HSCs, potentially allowing normalization of hematopoiesis in the absence or after mild conditioning.
Difficulties in the collection of sufficient numbers of HSC from FA patients and the use of sub-optimal transduction protocols with gammaretroviral vectors limited the success of FA gene therapy trials conducted 10 years ago in the USA. EuroFancoLen innovative approach is to develop for the first time an efficient and safe gene therapy of FA based on two recent innovations: 1) Discovery of potent HSC mobilizers, such as plerixafor Ans 2) Development of a new lentiviral vector by members of this Consortium, designed as Orphan Drug by the Europan Commission in December 2010: The main objective of this project is, therefore, the development of a multicentric Phase I/II gene therapy trial for FA-A patients, based on the genetic correction of plerixafor+G-CSF mobilized HSCs with the novel lentiviral vector, accompanied by comprehensive and groundbreaking safety and efficacy patient monitoring estudies.
The Spanish cell therapy network (TerCel) is a collaborative research project organized by the Spanish National Institute of Health Carlos III that started in 2003, to promote research in cell therapy and translate the scientific advances in this field to the clinic. Based on a multidisciplinary approach and the interaction and cooperation between 33 groups of basic and clinical scientists across Spain, the main objective of TerCel is to develop new medical therapies based on the use of stem cells for cardiovascular diseases, neurodegenerative diseases and osteo-articular, immune-hematologic and metabolic diseases.
Rare diseases, affecting around 3 million Spanish people, are a social and health problem of the first order. The Centre for Biomedical Network Research on Rare Diseases (CIBERER), a network structure set up at the initiative of the Instituto de Salud Carlos III, pools and furthers the excellence research done in this country with the aim of finding diagnoses and therapies for those affected as quickly as possible.
Charles River Laboratories
For more than 60 years, Charles River has been helping our global partners accelerate drug discovery and development by providing them with high-quality research models and preclinical and clinical support services. Our offerings span the entire drug development process, from discovery through market approval, providing our clients a seamless partnership throughout.
GenoSafe is a Contract Services Organization and European Leader in evaluation of the safety, quality and efficacy of Gene and Cell therapy products. From research stages to clinical phases, we propose a real partnership in the conception, the development, the validation of analytical methods and in performing studies in strict compliance with regulatory requirements.
We focus on the following major axes:
- Customised molecular studies: development and validation of qPCR and RTqPCR-based methods, biodistribution and shedding studies
- Customized studies for characterization of immune responses
- Quality controls specific to viral vectors ("tox" and GMP batches)
- Clinical trial-patient follow-up: functional testing, shedding, viral safety and immunomonitoring
GenoSafe is GLP (level A) compliant.
BioReliance was acquired by Sigma-Aldrich Corporation, in January, 2012 to supply customers access to a powerful, single-point provider whose products and services span the drug discovery, development and commercialization pipeline. As part of the SAFC business unit, BioReliance is a leading provider of cost-effective contract services, offering more than 1,000 tests or services related to biologics safety testing, specialized toxicology and animal health services. BioReliance provides outsourced services to thousands of customers from most healthcare disciplines and is the largest provider of safety testing services focused on the rapidly growing biologics sector of the pharmaceutical and biotechnology industries.
BioReliance has over 700 employees and has laboratory operations in Rockville, MD and Scotland
uniQure is the world leader in the development of human gene based therapies. uniQure's Glybera, a gene therapy for the treatment of lipoprotein lipase deficiency has been approved in the European Union, and is the first approved gene therapy in the Western world. uniQure's product pipeline of gene therapy products in development comprise hemophilia B, acute intermittent porphyria, Parkinson's disease and SanfilippoB. Using adeno-associated viral (AAV) derived vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design the world's first stable and scalable AAV manufacturing platform, validated in the approval process of Glybera. This proprietary platform can be applied to a large number of rare (orphan) diseases caused by one faulty gene.
ANDALUSIAN INITIATIVE FOR ADVANCED THERAPIES
Who we are: The Andalusian Initiative for Advanced Therapies(IATA) is a publicly funded organization created by the Regional Government of Andalusia (Junta de Andalucía) to promote activities of R&D&i in the fields of Cell Therapy and Regenerative Medicine, Clinical Genetics and Genomic Medicine and Nanomedicine in orderto foster both cell and gene technologies and therapies for regenerative medicine applications.
We are part of the Andalusian Public Healthcare System that offers complete health services to about 9 million people and comprises, among other infrastructures: 45 Hospitals, 1,500 Primary Care Centres, several Research Centres and Institutes, a network of 10 GMP facilities to manufacture gene and cell based therapies, a Genomics and Bioinformatics Platform and a Biobank storing more than 670.000 samples from patients and normal controls –including hiPS and hES cells lines. We act as an interface between academia (hospitals, universities and other research institutions) and the industry (biotechnological and pharmaceutical companies) to facilitate translational research and technology transference.
What we offer: Being part of such a multidisciplinary system makes the IATA an ideal partner offering comprehensive support for translational research. Moreover, being the promoters of 22 clinical trials with different gene and cell-based therapies (3 of them in phase III) we can offer regulatory expertise and advisory support. In fact, we organize a unique European Master Degree in Manufacturing of Advanced Therapies Medicinal Products.
What we seek: We seek alliances among the academic world, research institutions, health centres, patients' associations, SMEs and the pharmaceutical industry. Furthermore, we are looking to establish collaborations based not only on offering services but specifically in creating partnerships within a risk-benefit sharing model to enhance and accelerate the development of new products.
ATMI, (Nasdaq: ATMI) is a global leader in enabling process materials and process technology for semiconductor, display and life science industries. At ATMI, process ingenuity unleashes new process possibilities for customers.
La empresa Miltenyi Biotec desarrolla, fabrica y vende más de 1000 productos y servicios, en particular en los campos de biología celular, inmunología, medicina regenerativa, y biología molecular.
La compañía está comprometida con el avance del entendimiento científico y de la medicina suministrando productos y servicios para la investigación biomédica y la terapia celular.
Nuestro objetivo principal es progresar en los campos del entendimiento científico y de la medicina proporcionando herramientas para la investigación biomédica y la terapia celular.
El área y ámbito de actuación de Miltenyi Biotec es la terapia celular y la investigación en los campos de regeneración de órganos, modulación inmune y trasplante.
REGENER-AR (Bringing Regenerative Medicine into the market: Allogeneic eASCs Phase IB/IIA clinical trial for treating Rheumatoid Arthritis) is a clinical translational collaborative project which aims at developing a broadly available and clinically applicable treatment for Rheumatoid Arthritis (RA) by exploiting the biology of living human expanded allogeneic mesenchymal adult stem cells extracted form adipose tissue (eASCs).
To accomplish this goal we will test advanced cellular therapy through a phase Ib/IIb clinical trial in order to define the safety and feasibility of the systemic administration of allogeneic eASCs in patients with RA. We will also advance in the difficulties and barriers that prevent cell therapy to become a largely available product in the market by facing several issues related to the production scale-up, effective clinical treatment and regulatory affairs compliance.
GE Healthcare Life Sciences provides tools for drug discovery, biopharmaceutical manufacturing and cellular technologies, so research scientists worldwide can be more productive, effective and creative.
Our vision is to be the start-to-finish bioprocessing solution provider, the partner of choice in cell and protein research and the leader in life sciences services.
Building on our broad expertise across life sciences, we are firmly committed to help researchers understand life at a molecular level and develop solutions that can prevent, diagnose, treat, and cure disease.
Created in 1958 by a group of patients and their families, and recognized as being of public utility in 1976, AFM (French Muscular Dystrophy Association) has a single objective : to defeat neuromuscular diseases which are devastating muscle-wasting diseases. It has set itself two missions : curing neuromuscular diseases and reducing the disabilities they cause.
REGENX BioSciences is leading the effort to translate promising gene delivery applications into a pipeline of next generation personalized therapies for a range of severe diseases with serious unmet needs. Our proprietary NAV™ technology is based on NAV vectors, the next generation of recombinant adeno-associated virus (rAAV) vectors, including NAV rAAV8, NAV rAAV9, and NAV rAAVrh.10. We believe that the NAV technology represents the potential promise of curing the root cause of disease rather than the symptoms, and we are committed to establishing best in class standards for our NAV vectors.
Celgene is a global biopharmaceutical company committed to improving the lives of patients with certain types of cancer and other rare incurable diseases.
Our mission is to deliver truly innovative and life-changing drugs for our patients. As a company we work to build a major global biopharmaceutical corporation while focusing on the discovery, the development, and the commercialization of products for the treatment of cancer and other severe, immune, inflammatory conditions.
There are more than 300 clinical trials at major medical centers using compounds from Celgene. Investigational compounds are being studied for patients with incurable hematological and solid tumor cancers, including multiple myeloma, myelodysplastic syndromes, chronic lymphocyte leukemia (CLL), non-Hodgkin's lymphoma (NHL), myelofibrosis, small cell lung cancer and prostate cancer.
apceth is a clinical stage biopharmaceutical company in the Munich area focused on the development of native and genetically modified cell therapeutics ("Advanced Therapy Medicinal Products", ATMPs). We combine the principles of adult stem cell biology with pioneering technologies and the highest standards for GMP manufacturing and quality management, according to European and international guidelines.
We offer contract GMP manufacturing and related services to customers around the world, using our expertise, high standards and state-of-the-art GMP/BSL2 facilities of more than 600 m2 of cleanroom area (class ISO 8, ISO 7, ISO 5), quality control units and R&D laboratories. Our manufacturing license covers native and genetically modified cell therapeutics, according to §13 and §20b of the German Medicines Act (AMG).
We are the first company worldwide to receive approval for a phase I/II clinical trial with genetically modified mesenchymal stem cells for the treatment of advanced gastrointestinal cancer (start 10/2013). In parallel, our first clinical phase I/II trial for the treatment of critical limb ischemia with our somatic cell therapeutic is currently ongoing.
Genetrix, constitutes a group of biotech companies that develop and commercialize innovative products in the following markets:
- Tools to amplify, repair and sequence DNA, which are essential for the main Molecular Biology techniques used in the lab and playa key role in the up-and-coming Personalized Medicine field.
- Cell therapy, based on adult stem cells,which has proved to have enormous potential to tackle diseases that currently do not have established therapeutic treatment, such as myocardial infarction, rheumatoid arthritis o Parkinson’s disease.
The Genetrix Group structure has been designed to:
Transform academic results into:
- Innovative sanitary products and services
- Molecular Biology tools with global market potential
Focus on high value investments, with the support of relevant industrial and financial partners for every project.
NIMGenetics is a company of biomedical technology dedicated to the design, innovation, development and commercialization of last generation products and services for genetic diagnostic. Genomic data analysis and interpretation by NIMGenetics team is one of our strengths and a key element in the company value chain.
NIMGenetics team uses two of the most powerful and state-of-the-art technologies: array CGH, with our own designs manufactured by Agilent Technologies and Next Generation Sequencing using Ion Torrent. These technologies are a hundred times more potent and accurate than traditional karyotype. We use them for: Prenatal and Postnatal Diagnostics, Oncology, Cardiology and Stem Cells Quality Control. NIMGenetics also provides support services for R&D institutions.
NIMGenetics was created by Dr. Cigudosa (CNIO), Dra. Álvarez (CNIO) and Dr. Samper (CNIC). This team is a national reference in molecular and diagnostic cytogenetics as well as in stem cells biosafety. NIMGenetics has 14 employees, 7 of them Ph.D in biomedicine.
NIMGenetics is an authorized Center of Analytic Diagnostics with a Genetic Unit, and has the accreditation as Agilent Certified Service Provider (CSP) in Spain.
The Project AIPGENE is a novel gene therapy treatment for Acute Intermittent Porphyria stems from the need to investigate new approaches for AIP disease. This allows us to offer the possibility of a novel treatment to mitigate AIP, which might cover the needs of these patients, offering them a better quality of life.
This Collaborative Project involves seven partners from four different European countries and it is co‐financed under the European Commission’s 7th Framework Programme for Research. The project is co‐ordinated by the Center for Applied Medical Research (CIMA), a European Center of excellence for Research, established in 2004 and located in Pamplona, Spain. The other partners implicated are uniQure (Netherlands), Clínica Universidad de Navarra (Spain), the Karolinska University Hospital (Sweden), the National Center for Tumor Diseases (Germany), Digna Biotech (Spain) and the Hospital 12 de Octubre (Spain). The principal aim of the AIPGENE project is to develop the clinical potential of the orphan drug AAV5‐AAT‐PBGDfor the treatment of the rare disease AIP. Successful completion of this project will represent an important advance in the clinical and therapeutic management of AIP and of metabolic liver diseases in general, as well as in our understanding of their physiopathology.
CellCAM “A New generation of safer and more efficient cellular treatments” is a consortium of the Biomedicine R+D Program 2010 of the Autonomous Community of Madrid. The consortium is composed by 8 groups from different research centres of Madrid with international prestige, coordinated by Dr. Juan A. Bueren (Ciemat) in which also participate Dr. A. Bernad (CNIC), Dr. J.G Castro (BIOC-ISCIII), Dr. F. Fernández-Avilés (HGUGM), Dr. M. Del Río (Uc3M), Dr. D. García Olmo (idiPAZ), Dr. A. Zapata (UCM), Dr. M. Ramírez (FIBHUJNS) and the two associated companies TIGENIX S.A.U. in research activities and Janus Developments S.L. that carries out research management activities. The main objective of this Consortium is to consolidate the translational research in cellular therapy to develop treatments for different diseases with a potential curative treatment by these strategies.
A reliable, single-source research partner providing high-quality reagents, technologies and industry-leading service to the life science and high technology markets. Scientific knowledge, innovation, high quality products and reliable delivery that support consistent, predictable results and save researchers time, are our strenght. The Company operates in 35 countries and sales in 160 countries whose objective is to provide excellent service worldwide.
Global solutions for research and manufacturing customers, with our brands :
· Sigma® Life Science : kits and reagents including genomics, functional genomics, proteomics and cell biology.
· Aldrich® Chemistry : organic and inorganic chemicals, building blocks, reagents, advanced materials and stable isotopes.
· Fluka® Analytical : Chemicals and reagents for analytical, organic and biochemical and pharmaceutical research and industries.
· Supelco® Analytical : Chromatography columns and related tools. · SAFC®: Customized services and manufacturing.
TiGenix is a leading European cell therapy company with a commercial product and a strong clinical stage pipeline of adult stem cell programs. The companys lead product, ChondroCelect®, for cartilage repair in the knee, is the only approved cell-based product in Europe, and is currently being launched across Europe. TiGenixs adipose derived allogeneic stem cell platform has been extensively validated. TiGenix is based out of Leuven (Belgium) and has operations in Madrid (Spain), and Sittard-Geleen (the Netherlands).