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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.
Oxford BioMedica (LSE:OXB) is a leading gene and cell therapy group focused on developing life changing treatments for serious diseases. Oxford BioMedica and its subsidiaries (the "Group") have built a sector leading lentiviral vector delivery platform (LentiVector®), which the Group leverages to develop in vivo and ex vivo products both in-house and with partners. The Group has created a valuable proprietary portfolio of gene and cell therapy product candidates in the areas of oncology, ophthalmology and CNS disorders. The Group has also entered into a number of partnerships, including with Novartis, Bioverativ, Sanofi, Axovant, Orchard Therapeutics, Boehringer Ingelheim/UK Cystic Fibrosis Gene Therapy Consortium/Imperial Innovations, GC LabCell and Immune Design, through which it has long-term economic interests in other potential gene and cell therapy products. Oxford BioMedica has world class facilities and capabilities to support pre-clinical, research and bioprocessing development through to GMP production and supply of commercial and clinical trial materials. The production activities are focused on the manufacture of lentiviral vectors from human cell lines in large scale serum free suspension cultures. Oxford BioMedica is based across several locations in Oxfordshire, UK and employs more than 320 people. Further information is available at www.oxfordbiomedica.co.uk.
MolMed S.p.A. is a medical biotechnology company focused on research, development and clinical validation of novel anticancer therapies. MolMed’s pipeline includes antitumour therapeutics in clinical and preclinical development: Zalmoxis® (TK), a cell-based therapy; NGR-hTNF, a novel therapeutic agent for solid tumours; CAR-CD44v6, an immuno-gene therapy project; MolMed also conducts cell and gene therapy projects in collaboration with third parties, offering resources and expertise covering preclinical to Phase III trials activities. MolMed is listed on the main market (MTA) of the Milan stock exchange managed by Borsa Italiana
Intellia Therapeutics is a leading gene editing company, focused on the development of proprietary, potentially curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course. Our combination of deep scientific expertise and clinical development experience, along with our leading intellectual property portfolio, puts us in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Intellia was named as one of the top 10 biotech start-ups by Nature Biotechnology. In September 2015, Intellia was named a “Fierce 15” biotech company by FierceBiotech.
Adverum is a clinical-stage gene therapy company targeting unmet medical needs in serious rare and ocular diseases. Adverum has a robust pipeline that includes product candidates designed to treat rare diseases alpha-1 antitrypsin (A1AT) deficiency and hereditary angioedema (HAE) as well as wet age-related macular degeneration (wAMD). Leveraging a next-generation adeno-associated virus (AAV)-based directed evolution platform, Adverum generates product candidates designed to provide durable efficacy by inducing sustained expression of a therapeutic protein. Adverum has collaboration agreements with Regeneron Pharmaceuticals to research, develop, and commercialize gene therapy products for ophthalmic diseases and Editas Medicine to explore the delivery of genome editing medicines for the treatment of inherited retinal diseases. Adverum’s core capabilities include clinical development and in-house manufacturing expertise, specifically in process development and assay development. For more information please visit www.adverum.com.
Brammer Bio is a best-in-class viral vector contract development and manufacturing organization (CDMO) for companies developing gene-modified cell therapies and in vivo gene therapies. Brammer offers end-to-end CDMO services from Phase I/II in Florida through commercial manufacturing in Massachusetts. Brammer Bio provides clinical and commercial supply of viral vectors for in vivo gene and ex vivo modified-cell based therapies, process and analytical development, and regulatory support, enabling large pharma and biotech clients to accelerate the delivery of novel medicines to improve patient health. The Brammer team consists of extraordinary employees focused exclusively on serving the gene therapy needs of clients and their patients. We are Helping to Cure.®
Development of an innovative gene therapy platform to cure rare hereditary muscle disorders (#667751; ~€6 million; 4 years), is an EU-wide-H2020 research project funded by the European Commission. MYOCURE aimed to develop a novel, innovative and clinically-translatable one-treatment platform for patients suffering from rare inherited muscle diseases, specifically focusing on myotubular myopathy (MTM) and glycogen storage disorder (GSD) type II. The objectives of MYOCURE are: (1) To boost gene transfer and expression using muscle-specific vectors, (2) To minimize undesirable immune reactions and to validate the efficacy and safety of the innovative vector platform technology in preclinical MTM and GSD II animal models, (3) To develop a scalable GMP-like manufacturing process for subsequent GLP toxicology studies, (4) To apply for an orphan drug designation for the innovative advanced therapy medicinal product (ATMP), (5) To consolidate a Phase I gene therapy clinical trial in patients suffering from MTM and GSD
Rocket Pharma is a clinical-stage gene therapy company with a multi-platform approach for the treatment of patients with devastating diseases. We leverage LV and AAV transduction strategies towards first-in-class programs. Hallmarks of Rocket’s vision include: 1) a high threshold for the selection of quality programs, 2) leverage of deep industry know-how through a world-class team and manufacturing partnerships and 3) a laser focus on optimizing and innovating gene therapy product parameters through a seasoned scientific approach that de-risks programs as they enter clinical trials.www.rocketpharma.com
Audentes Therapeutics is a clinical stage biotechnology company focused on developing and commercializing gene therapy products for patients living with serious, life-threatening rare diseases. Audentes is currently developing four product candidates, including AT132 for the treatment of X-Linked Myotubular Myopathy (XLMTM), AT342 for the treatment of Crigler-Najjar Syndrome, AT982 for the treatment of Pompe disease, and AT307 for the treatment of the CASQ2 subtype of Catecholaminergic Polymorphic Ventricular Tachycardia (CASQ2-CPVT). We are a focused, experienced and passionate team committed to forging strong, global relationships with the patient, research and medical communities.
Sarepta Therapeutics is a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicines to treat rare neuromuscular diseases. The Company is primarily focused on rapidly advancing the development of its potentially disease-modifying Duchenne muscular dystrophy (DMD) drug candidates and is proud to support the 2018 AANEM Annual Meeting. For more information about Sarepta.
Biopharma Excellence is THE consulting company for biopharmaceutical drug development and regulatory strategies. Biopharma Excellence supports clients to bring their ideas to approval. The Biopharma Excellence team comprises experienced experts with proven track records coming from agencies, industry or service providers. We are THE experts for advanced therapy medicinal products (ATMP) such as cell, tissue and gene therapies. Biopharma Excellence has supported numerous unique ATMP development projects by preparing and executing tailored strategies in order to speed up the process from early-stage development to initiation of clinical studies up to marketing authorization.
Lonza offers world-class technology platforms in the areas of GMP cell culture and viral-based therapeutic manufacturing, custom bio-therapeutic culture media, a large selection of primary and stem cells and a full line of custom bioassays. Our extensive experience in cell therapy process optimisation and scale-up innovation helps clients to safely and effectively advance their products through all phases of the commercial pipeline and maximise their return on investment. Our new viral-based therapeutics group provides viral vaccine manufacturing as well as viral vector mediated gene therapies. Our staff can design, develop and implement a manufacturing process that meets your autologous or allogeneic therapeutic applications.
BioNTech Innovative Manufacturing Services – EXPERTS IN CELL AND GENE THERAPY MANUFACTURING BioNTech Innovative Manufacturing Services (IMFS) is a German Contract Development and Manufacturing Organisation specialized in the industrialization of cell and gene therapy products (viral vectors, cells and ivt mRNA). Based on extensive expertise in scientific, technical and regulatory prerequisites, we develop and manufacture your products in a safe and cost-efficient way in our state-of-the-art GMP facility. We offer a complete service spectrum from process development through clinical trial to in-market supply. All services are fully integrated and supervised by our QA department ensuring efficient and compliant manufacturing in our state-of-the-art GMP facility.
Yposkesi is the largest European CDMO for gene therapy vector manufacturing. Created in November 2016 in Corbeil Essonnes (France) as a spin off from the world-class gene therapy pioneer Genethon, Yposkesi provides a full-service offer covering BioProcess development (USP & DSP), from small/pilot to large production scale,analytical development, GMP manufacturing of clinical lots of lentiviral vectors and regulatory support.
Its current facility consists in a 50,000 sq ft building, operating 4 manufacturing suites for bulk drug substance, and 2 Fill & Finish suites. This capacity will be expanded in 2021 with a large scale facility (50,000 sq ft) equipped with 2,000L bioreactors, designed for commercial production and compliant to EMA and FDA. Capitalizing on more than 25 years of expertise, Yposkesi significantly invests in innovation in bioprocessing to cost-effectively deliver on high quality projects.
As part of Merck KGaA, Darmstadt, Germany, BioReliance® Services is a key component of the life science services portfolio and the leading provider of contract services in the biopharmaceutical industry. BioReliance’s comprehensive and integrated services support every phase of the testing, development and manufacturing process. With locations worldwide, BioReliance offers more than 1,000 tests and complementary services related to biologics safety testing and specialized toxicology. Our clients include the world’s top producers of traditional pharmaceuticals, medical devices and chemicals, as well as biopharmaceuticals
FinVector is a world leader in the research and development of Viral-Based Gene Therapy products, with state-of-the-art facilities and a highly experienced scientific team working in the gene therapy market. We deliver a tailored service to meet and exceed our clients’ needs, and use our scientific expertise and industry knowledge to help clients take viral-based products from the pre-clinical phase, through clinical trials and to the market.
VIVEbiotech is a company fully specialized in lentiviral vectors with two areas of expertise: 1. GMP CDMO: GMP Contract Development and Manufacturing Organization specialized in lentiviral vector bioprocess development (from very early stages) and GMP manufacturing 2. Innovation: VIVEbiotech´ strategy is focused on providing a response to the main hurdles that currently exist within the gene therapy field: a. The need for cost-effective processes. b.The enhancement of the safety profile of viral vectors: worldwide licensed non-integrative episomal stable lentiviral vector (LENTISOMA). VIVEbiotech is currently working with Companies based both in Europe and USA being our main aim being flexible in terms of technical adaptation and slots availability.
Vivet Therapeutics is a gene therapy biotech company with headquarters in Paris, France, dedicated to the research, development and future commercialization of gene therapy products for inherited liver disorders with high medical need.
Recently named as Fierce 15 biotech company, Vivet is committed to make a significant difference for patients facing rare metabolic diseases, with Wilson’s disease being our lead indication. Vivet has a strong presence in Spain through its research subsidiary and its strategic collaboration with Fundación para la Investigación Médica Aplicada (FIMA) at the Centro de Investigación Medica Aplicada (CIMA, University of Navarra, Spain), a leading research organization in the field of Gene Therapy. Vivet also has licensed the use of novel proprietary AAV vector gene therapy technology to treat metabolic diseases and certain patent-protected Anc80 AAV gene therapy vectors from Massachusetts Eye and Ear (MEE), a teaching hospital of Harvard Medical School, Boston. Anc80 is a next-generation gene therapy technology designed to increase gene expression levels in the liver.
Paragon Bioservices is an award-winning Contract Development & Manufacturing Organization (CDMO). Our aim is to build strong client partnerships focusing on transformative technologies, including oncology, immunotherapies (CAR-T cell therapies and oncolytic viruses), new generation vaccines (VLPs) and gene therapies (AAV). Paragon’s cGMP facilities include microbial and mammalian suites, aseptic fil-finish and full-segregated virus facilities.
Responsibility to our clients, a passion for science and our collective need to contribute to better public heath—is what keeps us motivated and excited about the work that we perform. We are driven by our commitment to provide exceptional quality scientific performance and customer service.
With over 25 years in the business, Paragon’s scientists, engineers, quality systems personnel and project managers have many years of experience working with biologics—from research and process development services to GMP manufacturing for clinical trials and eventual commercial launch.
Delphi Genetics, located in Belgium, is a biotechnology company specialized in genetic engineering with a deep expertise in plasmid DNA and its applications. Certified GMP we provide technological and flexible biomanufacturing solutions.
Delphi Genetics is involved in several DNA vaccines and gene therapy programs, including the development of CAR-T and collaborates on projects at all stages of development, from R & D to clinical phases. Our complete, GMP-compliant, flexible and single-use process for plasmid DNA manufacturing allows us to provide R&D, High Quality and GMP grade plasmid DNA from a few mg up to several grams in our dedicated facility.
To support this manufacturing activity, Delphi Genetics also offers a proprietary "antibiotic-free" technology, STABY®. The STABY® technology is implementable in all plasmid DNA and provide a better plasmid stabilization, a production yield increase and a safer way to manufacture plasmid DNA without antibiotic resistance gene following regulatory agencies recommendations.
Novasep is a CDMO specialized in viruses & viral vectors production for tox, clinical and commercial phases. Novasep offers global and integrated services from process development to commercial manufacturing. For more than 15 years, we have acquired experience in manufacturing viral vectors (lentivirus, AAV, ADV…) for gene therapy and vaccination. Our investment in new commercial assets, addressing both drug substance and drug product manufacturing processes, can help to contribute to your project’s success. Come and meet us at booth 33.
Amicus Therapeutics is a global biotechnology company at the forefront of therapies for rare and orphan diseases. The Company has a robust pipeline of advanced therapies for a broad range of human genetic diseases. As you can see from our website, we have a real interest in Gene Therapy and feel that we can partner with ESGCT on educating attendees of who Amicus is and what Amicus is working on.
uniQure is delivering on the promise of gene therapy, single treatments with potentially curative results. We have developed a modular technology platform to rapidly bring new disease-modifying therapies to patients treat patients with CNS, liver/metabolic and cardiovascular diseases. We are advancing a focused pipeline of innovative gene therapies and have entered late-stage clinical development in our lead indication, hemophilia B, and have established preclinical proof-of-concept in Huntington’s disease. www.uniqure.com
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