ESGCT is very grateful for support from our partners.
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.
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.
SPP 1230 is an interdisciplinary, multicentric Research Priority Programme of the German Research Council (DF G) with the mission to investigate “Mechanisms of gene vector entry and persistence”. The projects investigate basic mechanisms of cell entry, episomal maintenance or chromosomal insertion of transgenes, and the cellular and systemic responses to genetic cell modification. The overall aim of this network is to improve efficiency, predictability and biosafety of genetic therapy with a focus on hematopoietic cells.
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.
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.
PeproTech was established in 1988 by a group of scientists who decided to focus their efforts on the development and production of recombinant cytokines for life-science research.
Today, PeproTech is a world leader in supplying high quality cytokine products including E. coli, insect, and mammalian cell-derived recombinant proteins, their monoclonal/polyclonal antibodies, ELISA development kits, and other cytokine-related reagents.
These products are distributed worldwide through customer-service offices in the US, UK, France, Germany, Israel, Korea, China, Japan, Brazil, and Mexico.
We plan to maintain the highly competitive price of our products and to expand their distribution to Australia, New Zealand, and other locations. To keep up with current research and growing demand we have recently expanded our production capabilities to include a 5000 sq. ft. animal-free manufacturing facility. A key component in keeping up with current scientific advancements has been our collaboration with researchers in both academia and industry who provide us with suggestions necessary to continuously expand our product pipeline. We encourage you to convey to us your particular research needs and promise to do our best to fulfill them.
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.
It is an Association of motivated and informed parents and patients who share responsibilities within ELA and have united their efforts in order to fight leukodystrophies and myelin diseases by establishing and respecting the following clear objectives:
- to help and support families affected by leukodystrophy,
- to stimulate the development of research thanks to the ELA Foundation created in 2005,
- to raise public awareness,
- to develop its work at international level.
ELA is thus a bridge between all forms of leukodystrophies and a family solidarity network.
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.
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.
Celogos (www.celogos.fr) is a privately-held biotech company that engineers innovative products, devices and services in cellular & tissular therapies for regenerative medicine. Celogos aims to identify, develop, register & commercialize cell therapy products which fill therapeutic gaps for unmet medical needs. Celogos works as an engineering organization coordinating the activities of the various actors participating in the elaboration of cell therapy products. Celogos will commercialize its products through collaboration with its mother company, HRA Pharma (www.hra-pharma.com). Celogos has entered a partnership with Rouen University Hospital and Inserm U905 to develop a cellular therapy for fecal incontinence. Contact: Christelle Doucet, PhD, R&D Director.
BrainCAV, a FP7 European Consortium aiming at generating new tools to understand and treat brain disease Formidable challenges remain to prevent and successfully treat brain diseases that devastate the lives of the affected individuals and too often their families. Traditional pharmacological approaches (i.e. drugs), as well as those using stem cells, made progress but their impact remain limited. Recent results from "gene therapy" trials where we use "DNA as a drug" gives us substantial hope. In spite of its great potential however, gene therapy also brings unique obstacles. BrainCAV is a FP7 European collaborative project aiming at generating new tools to understand and treat brain diseases. Coordinated by Eric Kremer at CNRS-IGMM Institute in Montpellier, BrainCAV’s objective is to bring a novel viral vector "from bench to bedside". Using the unique capacity of specific virus to preferentially infect neurons throughout the brain BrainCAV offers a translational approach that will help both researchers and clinicians understand fundamental neurobiology questions, disease progression and address therapy for brain diseases.To provide a proof-of-principle, the project initially tackles Sly Syndrome, a rare disease commonly affecting children, and Parkinson's disease, a well-known disease primarily affecting the aged population. BrainCAV was selected by the European Commission (Framework Program 7) and granted 3M€ out of a 4.5 M€ total budget on a 4 years period starting October 1st, 2008. By sponsoring a session on brain gene transfert and a special CNS Poster Award in the frame of ESCGT 2012 in Versailles, BrainCAV hopes to contribute and encourage beyond its own scope the interest in gene therapy and vectors development specifically dedicated to brain diseases.
BioOutsource is the world’s fastest growing provider of contract testing services to the biopharmaceutical industry. Our core offering includes a comprehensive range of services to support the testing of Biologics and Vaccines throughout their development. This is supported by BioOutsource’s highly experienced Management Team - derived from the former Senior Directors and Management at Q-One Biotech - who together provide over 125 years of contract testing experience. As a result we possess an unrivalled combination of commercial, regulatory and technical knowledge that enable us to truly stand out as the industry’s most unique and preferred testing partner.
At BioOutsource we don’t just perform contract testing services; we are committed to delivering service excellence. Our innovative approach which utilises tools such as BioWeb™ and In-life Auditing ensure that projects are managed efficiently, cost-effectively and delivered on-time. We understand the importance of our service to support our client’s critical testing requirements and as a result continually strive to provide a world class service.
Molecular Therapy - Nucleic Acids
New to NPG: Molecular Therapy—Nucleic Acids
Online. Open Access. High-Quality Research.
Molecular Therapy—Nucleic Acids is a sibling journal to the well-established high impact journal, Molecular Therapy. The journal will publish high quality basic, translational, and clinical research and cuttingedge reviews and commentaries targeted to the advances in oligonucleotide- and gene-based therapies. Molecular Therapy—Nucleic Acids is a peer-reviewed, fully open access, online journal. Molecular Therapy—Nucleic Acids is published on behalf of the American Society of Gene & Cell Therapy and NPG.
Sign up today for a Molecular Therapy—Nucleic Acids alert.
SAFC is a leading developer and manufacturer of critical raw materials and services that support the biopharmaceutical industry. Using unrivaled expertise, SAFC’s scientists identify and anticipate customer requirements and work to resolve the bioprocessing obstacles that can occur at every stage – from preclinical to commercialization. SAFC is known for safe, productive and consistent raw materials and services, providing customers with a greater feeling of confidence and control.
The SAFC portfolio includes proficiency in developing and manufacturing upstream and downstream solutions, single-use packaging and scale up services. It offers the widest selection of liquid media available to the market, encompassing custom media, supplements/feeds, buffers, water and reagents.
SAFC’s extensive liquid offerings are produced in state-of-the-art Centers of Excellence facilities that are cGMP compliant and employ fully validated processes. The Centers of Excellence act as an extension of a customers’ organization, designed to operationally deliver continuity of supply, consistency in raw material and process control and industry leading quality. Currently comprised of the company’s Broadway (St. Louis, Missouri), Lenexa, (Kansas) and Irvine (UK) facilities, these Centers address industry demands for efficiency, cost containment and increased regulatory compliance. With locations across the globe, SAFC is able to serve customer needs at source and without limitation. The global footprint of SAFC’s facilities offers customers supply chain security and mitigation of process delay risk through tailored solutions, ready-to-use formats, and on-time delivery.
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.
Cobra Biologics is a leading international clinical and commercial manufacturer of biologics and pharmaceuticals with three GMP approved facilities. We offer a broad range of integrated and stand-alone development services, stretching from cell line development through to the commercial supply of investigational medicinal product. We take pride in manufacturing excellence and being a trusted provider, delivering what we promise and helping our customers to develop drugs for the benefit of patients. Cobra Biologics provides manufacturing solutions to the biologics and pharmaceutical industry covering antibodies, recombinant proteins, viruses, DNA, cellular therapeutics and small molecules.
Institut Clinique de la Souris (iCS)
The Institut Clinique de la Souris (iCS) is an infrastructure of excellence in translational research and functional genomics. Genetic researches are conducted and supported combining generation and validation of mutant mice model and comprehensive phenotypic analysis (physiological -behavioral – anatomical). iCS provides scientists with a broad set of highly specialized mouse services. iCS is a reference center regarding animal models for human pathologies. It contributes to their better understanding and supports the generation of novel therapeutics. For more information about iCS, please visit our web site
Orgenesis is a development stage company with a novel therapeutic approach in the treatment of diabetes by correcting malfunctioning organs with new functional tissues created from the patient’s own existing organs. Orgenesis employs a molecular and cellular approach directed at converting liver cells into functional insulin-producing cells as a treatment for diabetes. This new therapeutic approach is called Autologous Insulin Producing (AIP) cell transplantation.