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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.
Spark Therapeutics, a fully integrated company, strives to challenge the inevitability of genetic disease by discovering, developing, and delivering gene therapies that address inherited retinal diseases (IRDs), neurodegenerative diseases, as well as diseases that can be addressed by targeting the liver, such as hemophilia. Spark Therapeutics has ongoing clinical trials investigating gene therapies in hemophilia A and B. SPK-8011 is in an ongoing, dose-escalation Phase 1/2 clinical trial as a potential one-time therapy for hemophilia A. The company retains full global commercialization rights to the SPK-FVIII program. SPK-9001, which has received both breakthrough therapy and orphan product designations by FDA, and access to the PRIority MEdicines (PRIME) Program by EMA, is in a Phase 1/2 clinical trial for hemophilia B and is being developed in collaboration with Pfizer. Our most advanced investigational candidate, with proposed trade name LUXTURNA™ (voretigene neparvovec), is currently under Priority Review with FDA for the treatment of biallelic RPE65-mediated IRD and has been designated as a drug for a rare pediatric disease. The MAA for LUXTURNA has been submitted to EMA for the treatment of vision loss due to Leber congenital amaurosis or retinitis pigmentosa caused by confirmed biallelic RPE65 mutations. LUXTURNA has received breakthrough therapy and orphan product designations from FDA and orphan product designations from EMA. The pipeline also includes SPK-7001 in an ongoing Phase 1/2 clinical trial for choroideremia.
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.
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.
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.
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.
Brammer Bio, a part of Thermo Fisher Scientific, is a leading viral vector CDMO for companies developing gene-modified cell therapies and in vivo gene therapies. Brammer provides process and analytical development, clinical and commercial supply of viral vector drug substance and drug product, and regulatory support, enabling our clients to bring novel medicines to market. Brammer is flexible to support every client’s unique product needs, timelines and regulatory pathways. Through recent investments in our world-class facilities and planned expansion, we are strategically positioned to support your immediate and long-term viral vector manufacturing needs.
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.
LumaCyte is an advanced biotechnology instrumentation company focused on driving innovation into the single cell analysis and biomanufacturing markets. We provide tools that enable cell biologists, biomedical researchers, as well as biopharmaceutical and biomanufacturing companies the opportunity to harness label-free multivariate data for identification, characterization, and sorting of cells of interest where the use of antibody or genetic labeling is not required.
Applications of LumaCyte’s Laser Force Cytology (LFC) platform include viral vector, vaccine and cell therapy (CAR T, TCR, and iPSC) R&D and manufacturing, cancer biology R&D, gene therapy, and pre-clinical drug discovery. Specific examples include rapid viral infectivity measurements to accelerate process and cell-line development for viral vector manufacturing as well as label-free phenotypic characterization of cell response and iPSC differentiation monitoring.
MeiraGTx (NASDAQ:MGTX) is a vertically integrated, clinical stage gene therapy company with four ongoing clinical programs and a broad pipeline of preclinical and research programs. MeiraGTx has core capabilities in viral vector design and optimization and gene therapy manufacturing, as well as a potentially transformative gene regulation technology. Led by an experienced management team, MeiraGTx has taken a portfolio approach by licensing, acquiring and developing technologies that give depth across both product candidates and indications. MeiraGTx’s initial focus is on three distinct areas of unmet medical need: inherited retinal diseases, severe forms of xerostomia and neurodegenerative diseases. Though initially focusing on the eye, salivary gland and central nervous system, MeiraGTx intends to expand its focus in the future to develop additional gene therapy treatments for patients suffering from a range of serious diseases. For more information, please visit www.meiragtx.com.
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
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.
Orchard Therapeutics is a fully integrated commercial-stage biotechnology company dedicated to transforming the lives of patients with rare diseases through innovative gene therapies.
Orchard’s portfolio of autologous ex vivo gene therapies includes Strimvelis, the first autologous ex vivo gene therapy approved by the EMA for adenosine deaminase severe combined immunodeficiency (ADA-SCID). Additional programs for primary immune deficiencies, inherited metabolic disorders and blood disorders include three advanced registrational studies for ADA-SCID, metachromatic leukodystrophy (MLD) and Wiskott-Aldrich syndrome (WAS), clinical programs for X-linked chronic granulomatous disease (X-CGD) and beta-thalassemia, as well as an extensive preclinical pipeline.
Orchard currently has offices in the U.K. and the U.S., including London, San Francisco and Boston.
Using our unique LentiVector® delivery platform, we have created a valuable portfolio of gene and cell therapy product candidates in the areas of oncology, ophthalmology and CNS disorders. We have strong partnerships with Novartis, Bioverativ (part of the Sanofi Group), Boehringer Ingelheim, the UK Cystic Fibrosis Gene Therapy Consortium and Imperial Innovations, Immune Design and Orchard Therapeutics, providing them with access to our intellectual property, state-of-the-art production facilities and expertise, and, in addition, we have licensed products and technology rights to Boehringer Ingelheim, Sanofi and Axovant. These partnerships provide us with multiple income streams, consisting of upfront milestone payments, development and production fees and potential royalties on future product sales. We plan to progress our wholly-owned products via spin-outs and out-licensing opportunities, while continuing to invest in our LentiVector® platform. We plan to continue our preclinical R&D to discover new potential products.
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas. REGENXBIO is currently developing four product candidates, including RGX-314 for the treatment of wet age-related macular degeneration (wet AMD), RGX-501 for the treatment of homozygous familial hypercholesterolemia (HoFH), RGX-111 for the treatment of mucopolysaccharidosis type I (MPS I) and RGX-121 for the treatment of mucopolysaccharidosis type II (MPS II).
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
Sarepta is at the forefront of precision genetic medicine, having built an impressive and competitive position in Duchenne muscular dystrophy (DMD) and more recently in gene therapies for 6 Limb-girdle muscular dystrophy diseases (LGMD), Charcot-Marie-Tooth (CMT), MPS IIIA, Pompe and other CNS-related disorders, totaling over 20 therapies in various stages of development. The Company’s programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. Sarepta is fueled by an audacious but important mission: to profoundly improve and extend the lives of patients with rare genetic-based diseases.
Synthego is a leading provider of genome engineering solutions. The company’s product portfolio includes software and synthetic RNA kits designed for CRISPR genome editing and research. With next-generation informatics and machine learning, Synthego’s vision is to bring precision and automation to genome engineering, enabling rapid and cost-effective research with consistent results for every scientist.
BIONTECH IMFS is a Contract Development and Manufacturing Organisation specialized in the industrialization of cell and gene therapy products (viral vectors, cells and ivt mRNA). For 20 years, BIONTECH IMFS has been developing and manufacturing retroviral vectors and cellular products for clinical supply in a variety of monogenetic diseases and different cancer indications.
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.
Editas Medicine is a leading genome editing company focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cpf1 genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. For the latest information and scientific presentations, please visit www.editasmedicine.com
ESTEVE is a global pharmaceutical group committed to the discovery, development and commercialization of innovative products to treat patients with unmet needs in the areas of neurology, rare disorders, and pain management. Founded in 1929, ESTEVE has an important commercial and industrial presence in Europe, US, Mexico and China, with capabilities covering the entire product life cycle including drug discovery, clinical development, manufacturing, distribution, and commercialization. ESTEVE’s commitment to innovation translates into a sustained investment of around 50 million Euros per year into developing specialty products. Within the Gene Therapy space, ESTEVE is currently developing 5 product candidates for the treatment of different types of Mucopolysaccharidosis, including EGT-101, an AAV-based product for the treatment of Mucopolysaccharidosis type III A (Sanfilippo A) which is currently in Phase I/II clinical trials.
Genethon, located in Evry, France, is a non-profit R&D organization dedicated to the design and development of gene therapy treatments for rare genetic diseases from research to clinical validation. It was created by the AFM-Telethon (French Muscular Dystrophy Association) which is its main funding source. 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 Audentes, Avexis/Novartis, Gensight Biologics, Orchard Therapeutics, Spark. Genethon was awarded the “Prix Galien France” in 2012.
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.
MaxCyte is a global cell-based medicines and life sciences company applying its patented cell engineering technology to help patients with high unmet medical needs in a broad range of conditions. The company leverages its Flow Electroporation® Technology to enable its partners across the biopharmaceutical industry to advance the development of innovative medicines, particularly in cell therapy, including gene editing and immuno-oncology. MaxCyte has placed its cutting-edge flow electroporation instruments worldwide, including with nine of the top 10 global biopharmaceutical companies, and has more than 55 partnered program licenses in cell therapy including more than 25 licensed for clinical use. With its robust delivery technology, MaxCyte helps its partners to unlock the full potential of their products.
Oxford Genetics mission is the creation of transformative systems and services to aid in the discovery and development of biologics, cell and gene therapies. A unifying theme across the portfolio is expertise in designing DNA and using this to optimise the expression of proteins, improving viral delivery systems and cell line engineering. Founded in 2011, Oxford Genetics is headquartered in Oxford, UK. Its products are sold directly and through a global network of distributors.
Thermo Fisher Scientific provides the quality products, services, and support you need to translate your gene and cell therapy from drug discovery through large-scale commercial production. We’re working alongside the scientific community to accelerate the pace of gene and cell therapy development and enabling scientists like you, to transform the world’s approach to health care.
Yposkesi is one of the largest Contract Development & Manufacturing Organizations (CDMO) in Europe for AAV and lentiviral vector production. A spin-off from the world-class gene therapy pioneer Genethon, Yposkesi capitalizes on more than 20 years’ expertise in biotherapeutic research to offer customers full integrated services; bioprocess development (USP & DSP), from small/pilot to large production scale, analytical development, GMP manufacturing of clinical batches of lentiviral and AAV vectors and regulatory support. Its current 50,000 sq ft (approx. 5,000m2) state-of-the-art facility designed for high efficiency houses four independent manufacturing suites for bulk drug substance and two Fill&Finish suites. Yposkesi is extending this capacity. By 2021 it will double its global footprint to 100,000 ft2 (approx. 10,000m2) with a second large-scale facility designed for commercial production and EMA and FDA compliance. Yposkesi invests significantly in innovation and bioprocessing to deliver on high quality projects, cost-effectively.
Aldevron serves the biotechnology industry with custom production of nucleic acids, proteins and antibodies. Featuring the largest and most modern GMP plasmid DNA manufacturing facility in the world, Aldevron provides thousands of clients with plasmids, RNA, gene editing enzymes and more for biological research projects from discovery to clinical trials and commercial applications. These products are critical raw materials and key components in commercially available drugs and medical devices, helping accelerate development of treatments for diseases such as cancer, infectious disease, pediatric disorders and rare diseases. Our collaborative approach and commitment to providing quality materials allow us to meet precise client requirements and provide innovative solutions to advance science. Aldevron headquarters is in Fargo, North Dakota USA, and has facilities in Madison, Wisconsin and Freiburg, Germany.
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.
Asklepios BioPharmaceutical, Inc. (AskBio) is a privately held, gene therapy platform company founded in 2001 and dedicated to improving the lives of children and adults with rare genetic disorders. AskBio’s gene therapy platform includes an industry-leading proprietary cell line manufacturing process and an extensive AAV capsid library. The company has generated hundreds of proprietary third generation gene vectors that have entered clinical testing, and maintains a portfolio of clinical programs across a range of indications.
Axon Medchem is a trusted supplier of high-value life science products, providing Axon Ligands™ as world wide recognized drug reference standards for pharmacological research. With more than 2000 excellent quality small molecule inhibitors and modulators targeting more than 850 biological targets, we aim to facilitate your scientific research and development.
Axon Medchem is also a leading European CRO in medicinal chemistry, specialized in contract research and high-quality synthesis of bio-active and/or drug-like molecules. We have the proven record in developing novel drug candidates and achieving excellence for a decade by providing our dedicated chemistry services for companies and research institutes around the world.
Batavia Biosciences is a biopharmaceutical CDMO with extensive experience in viral vector development and manufacturing projects. We offer our novel technologies and in-depth know-how in order to help our customers to complete preclinical phases at higher speed, reduced costs and increased success. The company focuses on the early stages of product development including cell line generation, upstream process development, purification development, product characterization and clinical manufacturing.
Biocair is the global specialist courier with over 30 years of dedicated experience in the pharmaceutical, biotechnology and life science sectors. Specifically, we provide dedicated, specialised logistics services – both the systems and the people – for the scientific sector and our services are the most comprehensive of its kind available on the market. Biocair’s global reach extends across Europe, Africa, Asia and the Americas.
The company has built up a unique, client-centric approach by employing scientists in front-line logistics positions and assembling a team of best-in-class industry experts in quality, cold chain and regulatory compliance amongst others. Biocair focuses on providing the most comprehensive service options available whilst delivering flexible, tailored, cost effective logistics solutions to all clients.
With six products on the market and a fully-integrated multinational organization in place, BioMarin is providing innovative therapeutics to patients with serious unmet medical needs. The company is also currently conducting a clinical trial of an AAV-based potential gene therapy for hemophilia A.
CELLINK is a global leader in 3D bioprinting, bioinks, and live-cell imaging technologies. CELLINK designs and develops bioprinting technologies that enable researchers to 3D print organs and tissue for applications that span industries, from pharmaceutical to cosmetic. Our patent-pending bioink is a biomaterial innovation that allows human cells to grow and thrive as they would in their natural environment. Researchers leverage our technologies to print liver, cartilage, skin tissues and more. In addition, our live-cell imager is designed for high-throughput analysis of multiple samples for rapid and ready-to-use results, thereby allowing researchers the ability to run powerful cell assays in parallel. Our single cell dispensing system can dispense virtually limitless combinations of cells, drugs, and growth factors in seconds for reproducible high-throughput downstream analysis. We invite you to join us on our journey to change the future of medicine!
CEVEC is a center of expertise for the production of biopharmaceuticals using a unique human cell-based production system. CAP-GT is a regulatory endorsed expression platform for scalable viral vector production. CEVEC has successfully developed CAP-GT suspension cell-derived viral packaging cell lines which enable better scale-up and competitive production costs when compared to adherent cell culture systems. CAP-GT suspension cell lines grow to high cell densities and show a broad viral propagation spectrum. Gene therapy vectors such as lentivirus (LV), adenovirus (AV) and adeno-associated virus (AAV) can be produced at industrial scale.
OVER 5 YEARS OF PARTNERSHIP
with hundreds of GMP studies performed through an integrated management scheme for recombinant, gene therapy, and vaccines products.
CLEAN CELLS CAPABILITIES
GLP/GMP certification for Quality Control and manufacturing
Bacterial, Eukaryotic (producer or packaging cells) and Viral Bank GMP manufacturing
IMP Manufacturing (cell therapy products)
Microbiological and viral testing
Identity – Karyotype - FISH
Adventitious virus testing
Specific contaminants detection (NAT methods)
Residual DNA/HCP quantitation
RcAAV & RCL
GMP certification for APIs and IMPs Manufacturing
Biopharmaceuticals Process Development
Biopharmaceuticals GMP Manufacturing (Drug Substance and Drug Product)
Bacterial and Yeasts Master/ Working Cell Bank GMP
Eukaryotic Master/Working Cell Bank GMP Manufacturing
Master/Working Virus Seed Stock GMP Manufacturing
Cell and Gene Therapy GMP Manufacturing
Fill and Finish
Quality Assurance Services
High flexibility and versatile structure
Dedicated Project Manager and single contact point
Highly qualified team
Covance Inc., the drug development business of Laboratory Corporation of America Holdings (LabCorp), is the world’s most comprehensive drug development company. We are dedicated to advancing healthcare and delivering Solutions Made Real® by providing high quality nonclinical, clinical, commercialization and informatics services to pharmaceutical and biotechnology companies to help increase the speed, precision and effectiveness of drug development.
We deliver unique perspectives by leveraging the largest volume of drug development and proprietary lab test data in the world. Covance is currently working on about half of all active clinical trials globally, has been involved in all top 50 best-selling drugs on the market and has supported more than 60% of all FDA-approved companion diagnostics. Covance offers the most experienced central laboratory in the industry, with deep biomarker expertise and extensive specialty testing capabilities supporting all therapeutic areas.
Together with our clients, Covance transforms today’s healthcare challenges into tomorrow’s solutions. Information on Covance’s solutions can be found by visiting www.covance.com.
cytena’s single-cell deposition technology isolates single, living cells automatically in a documented, viable and pure workflow. The single-cell printerTM and the x.sightTM instruments providing assurance of clonality by an image-series for each single cell, high cell viability like hand-pipetting, no cross-contamination by the use of sterilized disposable cartridges and are easy to use. 96 and 384 well plates can be used to deposit single-cells and single-bacteria with brightfield and fluorescence detection and assured clonality. So far, also most of the world's top 10 pharma companies successfully use our instruments for their cell line development.
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.
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.
GENEWIZ provides genomics services to over 120,000 users in 4,000 institutions worldwide, enabling researchers to advance their discoveries faster than ever before. Now a Brooks Life Sciences company, GENEWIZ leads the industry with our unique and proprietary sequencing and automation technologies backed by specialized experts in Next Generation Sequencing, GLP/CLIA regulatory-compliant services, Sanger Sequencing, and Synthetic DNA Solutions.
iBET is a private non-profit research-intensive institute dedicated to the development of innovative solutions for the production and purification of complex biopharmaceuticals. GenIbet Biopharmaceuticals is a GMP biopharmaceutical CDMO offering highly specialized GMP manufacturing and development services, including fill & finish.Together, GenIbet and IBET are able provide joint expertise covering a broad spectra of Biopharmaceuticals, including recombinant proteins, Vaccines, RNA, Live Microbial Products and Cell and Gene Therapy products. The portfolio includes Adenovirus, Adeno-Associated Virus, Retrovirus, Lentivirus, Baculovirus and Rhabdovirus, as well as a variety of stem cells such as induced pluripotent, human embryonic, mesenchymal, cardio and neural. In addition, fully owned Mass Spectrometry and GMP certified Analytical Services Units enable performing detailed quality control and characterization of our products.
Greiner Bio-One specialises in the development, production and distribution of high-quality plastic laboratory products.
The company is a technology partner for hospitals, laboratories, universities, research institutes, and the diagnostic, pharmaceutical and biotechnology industries.
The BioScience division of Greiner Bio-One ranks among the leading providers of specialised products for the cultivation and analysis of cell and tissue cultures.
Drawing on decades of experience with cryogenic sample storage, Greiner Bio-One also offers solutions for automated storage systems in biobanks.
In addition, we continue to utilise our expertise in the development and production of microplates for high-throughput screening, thereby allowing extremely fast
and efficient drug screening for both industrial and research applications.
The entire development, manufacturing and sales operations are controlled from the German headquarters of the BioScience division in Frickenhausen
For more than 30 years, IDT's innovative tools and solutions for genomics applications have been driving advances that inspire scientists to achieve their next breakthroughs. As the creator of the gBlocks® Gene Fragments, we share over 16 years of synthetic biology experience through innovative product development, real-time web-based order tracking, and experienced customer support staff. We have also developed proprietary technologies for creating best-in-class genomics solutions for CRISPR genome editing.
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.
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
NHS Blood and Transplant is a national organisation within the NHS dedicated to saving and improving lives.
Our Cellular and Molecular Therapies function offers broad experience and expertise in the provision and manufacture of cell and gene therapies through our national network of GMP grade and MHRA licensed facilities.
NHSBT partner with academic, commercial and NHS organisations supporting novel cell and gene therapy programmes from concept through to clinical trial, operating a ‘not for profit’ model.
We welcome expressions of interest for partnerships with organisations striving to develop the future of medicine in the advanced therapies space.
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.
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.
Polyplus-transfection applies its 15+ year expertise to the development of novel transfection solutions up to GMP grade for high yield transient protein and antibody production in CHO and HEK-293 cells, as well as for viral vector production for Gene and Cell Therapy (PEIpro product range).
PTC is a science-led, global biopharmaceutical company focused on the discovery, development and commercialization of clinically-differentiated medicines that provide benefits to patients with rare disorders. PTC’s ability to globally commercialize products is the foundation that drives investment in a robust pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need.
Sartorius Stedim Biotech is a leading international partner of the biopharmaceutical industry. As a total solutions provider, the company helps its customers to manufacture biotech medications safely, rapidly and economically. Headquartered in Aubagne, France, Sartorius Stedim Biotech is quoted on the Eurolist of Euronext Paris. With its own manufacturing and R&D sites in Europe, North America and Asia and an international network of sales companies, Sartorius Stedim Biotech has a global reach. The Group has been annually growing by double digits on average and has been regularly expanding its portfolio by acquisitions of complementary technologies. In 2018, the company earned sales revenue of €1,212.2 million, and currently employs some 5,800 people.
STEMCELL Technologies Inc is a privately-owned biotechnology company that develops specialty cell culture media, cell separation products and ancillary reagents for life science research. Driven by science and a passion for quality, STEMCELL delivers over 1500 products to more than 70 countries worldwide.
STEMCELL helps make sure your research works. We do it in many ways: in the creativity of novel products; in providing consistent, unfailing quality; and by standing with you, in person or by telephone, to provide one-on-one assistance to help you succeed. We do this because we love science as much as you do.
Takara Bio Inc. is the biotechnology company contributing to the health of humankind through the development of revolutionary biotechnologies such as gene therapy. We provide a wide range of life science reagents and kits under the Takara®, Clontech®, and Cellartis® brands. Since 2014, we have been focusing on the CDMO service, leveraging our know-how and expertise in developing gene therapy. We have been supporting numerous gene and cell therapy clinical trials through providing viral vectors, cell processing services and quality control testing.
Furthermore, among our GMP facilities, we have a specialized GMP facility for human pluripotent stem cell services in Sweden. This facility serves for clinical hES cell line derivation and various hiPS cell-related research including reprogramming, gene-editing and directed differentiation.
Takara Bio group devotes our efforts to meet the varied needs in ATMPs worldwide.
Touchlight AAV is a Joint Venture between AskBio and Touchlight Genetics, leaders in the AAV and DNA fields, to enable better AAV production. A union to provide the gold-standard DNA for AAV manufacture.
Touchlight AAV is advancing gene therapy by offering its synthetic, linear, double stranded DNA (dbDNA) as the new industry standard for transfection-based AAV production:
Research and Clinical grade dbDNA manufactured in our state-of-the-art facility in San Sebastian, Spain.
For more information, please visit: https://touchlightaav.com
Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.
For more information on Ultragenyx, please visit the Company's website at ultragenyx.com.
Univercells focuses on increasing the availability of affordable vaccines and biotherapeutics to address global health challenges. The company is developing turnkey solutions for a series of vaccines and biotherapeutics to be delivered at an affordable price. By relying on proprietary core technologies and a continuous process intensification approach, production is achieved with a smaller footprint, and significantly lower overall capital and operational costs.
Global company headquartered in Gosselies (Belgium), Univercells benefits from the support regional and national players as well as from the Bill & Melinda Gates Foundation, Global Health Investment Fund and other international players in the health and vaccine industry.
Viralgen Vector Core becomes the convergence point from experienced specialized organizations in biotechnology and life science fields, united with the aim of creating a CDMO in the sector of gene therapy and innovative medicines. Developing and manufacturing in order to contribute to the progress in health and welfare of people.
One of the key aspects for this exclusive technology to reach patients is the cGMP production for rAAV manufacturing in large batches. Askbio has been working in the field of gene therapy for more than 15 years and has developed its own cell line and manufacturing system. This allows large cGMP manufacturing of rAAV vectors with superior yield to others in 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 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.
Atara Biotherapeutics is a leading off-the-shelf, allogeneic T-cell immunotherapy company developing novel treatments for patients with cancer, autoimmune and viral diseases. Atara’s pipeline includes tab-cel® (tabelecleucel), which is in phase 3 development for patients with Epstein-Barr virus-associated post-transplant lymphoproliferative disorder (EBV+PTLD) as well as other EBV-associated hematologic malignancies and solid tumors, including nasopharyngeal carcinoma (NPC); T-cell immunotherapies targeting EBV antigens believed to be important for the potential treatment of multiple sclerosis; and next-generation chimeric antigen receptor T-cell (CAR T) immunotherapies for cancer as well as targets in other therapeutic areas. The company, founded in 2012, is co-located in South San Francisco (corporate headquarters) and Northwest Los Angeles (R&D and manufacturing) with a European headquarters established in 2018 in Zug, Switzerland.
LogicBio Therapeutics is a genome editing company focused on developing medicines to durably treat rare diseases in pediatric patients with significant unmet medical needs using GeneRide™, its proprietary technology platform. GeneRide enables the site-specific integration of a therapeutic transgene in a nuclease-free and promoterless approach by relying on the native process of homologous recombination to drive potential lifelong expression. Headquartered in Cambridge, Mass., LogicBio is committed to developing medicines that will transform the lives of pediatric patients and their families.
Innovations, designs and trademarks are a valuable asset in any business. They enhance your company’s distinguishing capacity, strengthen its competitive position and make it more attractive to investors. Therefore, there is every reason to give Intellectual Property a prominent place in your business strategy.
NLO has over 85 European patent and trademark attorneys, qualified in the Netherlands, Belgium, France, Europe and the United States. We are one of the largest IP consultancies in Europe with offices in the Netherlands (Amsterdam, The Hague, Eindhoven and Ede) and Belgium (Ghent and Mechlin). We support our clients by fortifying their innovations. With about 20 patent attorneys specialised in Biotechnology and Life Sciences, NLO has experienced dedicated teams, ensuring you to have access to the necessary expertise. Our client list includes multinationals, SMEs, universities, R&D and governmental organizations.
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.
Non-viral genetic therapy strategies for the treatment of ataxia telangiectasia
James Grey received the ESGCT-sponsored prize for best poster presentation at the British Society for Gene and Cell Therapy (BSGCT) Annual Conference, which took place from 19-21 June 2019. Read about his winning presentation here!
Gene therapy for thrombotic thrombocytopaenic purpura
Robyn Bell received the ESGCT-sponsored prize for best oral presentation at the British Society for Gene and Cell Therapy (BSGCT) Annual Conference, which took place from 19-21 June 2019. Read about her winning presentation here!
CRISPR-Cas9 genome editing - molecular scissors a bit too sharp?
Grégoire Cullot received the ESGCT-sponsored prize for best oral presentation at the Société Française de Thérapie Cellulaire et Génique (SFTCG) Thematic Day on Viral Vectors, which took place on 17 May 2019 in Paris. Read about his winning presentation here!
Dgkk as a new therapeutic target in the Fmr1-KO murine model of Fragile X
Karima Habbas received the ESGCT-sponsored prize for best poster at the Société Française de Thérapie Cellulaire et Génique (SFTCG) Thematic Day on Viral Vectors, which took place on 17 May 2019 in Paris. Read about her winning presentation here!
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