Patrocinadores de la SETGyC

Platino

bluebird bio

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

Oxford Biomedica

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

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.
 

MolMed

MolMed is a biotech company focused on innovative cell and gene therapies that can meet the therapeutic needs in the treatment of tumors and rare diseases, by combining scientific and research excellence with a clear and strong industrial projects.
MolMed is now structured on a dual business model, combining the development of onco-haematology proprietary pipeline with contract develop and manufacturing organization (CDMO), offering world class services and technologies to our clients.
With two approved manufacturing facilities (about 4.800 SQM) and top level expertise, MolMed offers platforms of lentiviral and retroviral vector manufacturing with different scales (48L up to 200L) and a consolidated processes in cells engineering (Hematopoietic Stem cells and T-cells).
 

MeiraGTx

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.

Orchard Therapeutics

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.

Intellia

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

Adverum is a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases. Adverum develops gene therapy product candidates designed to provide durable efficacy by inducing sustained expression of a therapeutic protein. ADVM-022, utilizes a propriety vector capsid, AAV.7m8, carrying an aflibercept coding sequence under the control of a proprietary expression cassette. ADVM-022 is administered as a one-time intravitreal injection, designed to deliver long-term efficacy and reduce the burden of frequent anti-VEGF injections, optimize patient compliance and improve vision outcomes for wet AMD and diabetic retinopathy patients. Adverum’s core capabilities include clinical development, novel vector discovery and in-house manufacturing expertise, specifically in scalable process development, assay development, and current Good Manufacturing Practices quality control.

Brammer Bio

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.
 

Myocure

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.
 

Rocket Pharma

Rocket Pharmaceuticals, Inc. is an emerging, clinical-stage biotechnology company focused on developing first-in-class gene therapy treatment options for rare, devastating diseases. Rocket’s multi-platform development approach applies the well-established lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapy platforms. Rocket's first two clinical programs using LVV-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, and Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal. Rocket’s first clinical program using AAV-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. Rocket’s pre-clinical pipeline programs for bone marrow-derived disorders are for Pyruvate Kinase Deficiency (PKD) and Infantile Malignant Osteopetrosis (IMO).
 

Audentes

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

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

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.
 

Biopharma Excellence

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.

NHSBT

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.

LumaCyte

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. 
 

Terumo BCT Europe

Terumo BCT, a global leader in blood component, therapeutic apheresis and cellular technologies, believes in the potential of blood and cells to do even more for patients than they do today. Terumo BCT’s Cell Therapy Technologies business enables researchers, developers and manufacturers to create next-generation cell and gene therapies. We do this through flexible, automated solutions that help meet your evolving needs for reproducibility, quality and scale through the phases of development, from translational research to current good manufacturing practice (cGMP) for commercial manufacturing. One example, the Quantum® Cell Expansion System automates and optimizes cell culture to meet the needs of a variety of cell types in a consistent, controlled and functionally closed environment. We are also looking further downstream in the process to provide innovative solutions, like the FINIA® Fill and Finish System, to support your needs into the future.

Oro

Genethon

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

BD

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. Products/Services · 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 manufacturing

Human Gene Therapy

Established in 1990, Human Gene Therapy is the leading peer-reviewed journal publishing exceptional, multidisciplinary research embracing all aspects of gene therapy - from basic research to new technologies to clinical development. In 2020 the Journal will mark three impressive decades by unveiling a dramatic new redesign and a renewed editorial mission. Stop by booth 29 to pick up free copies of Human Gene Therapyexplore a suite of CRISPR and gene therapy publications, and learn more about the exciting new changes coming next year. Human Gene Therapy serves as the Official Journal of ESGCT.

Editas Medicine

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

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. 

Lonza Pharma & Biotech

At Lonza Pharma & Biotech, we provide contract development and manufacturing services that enable pharma and biotech companies to bring medicines to patients in need.  From the building blocks of life to the final drug product, our solutions are created to simplify your outsourcing experience and provide a reliable outcome when you expect it. Our extensive track record includes commercialization of pioneering therapies and manufacturing of a wide variety of biological and chemical drugs. We continuously invest to solve not just the current, but also the future challenges. Together, let’s bring your next medicine to life.
 

Oxgene

OXGENE find solutions to seemingly impossible problems. We develop new technologies in mammalian cell engineering along the whole spectrum from discovery science to engineering at scale. Our unique combination of precision engineering and breakthrough science with advanced robotics and bioinformatics accelerates the rational design, manufacture and processing of complex biologics.
We work at the very edge of impossible to solve the problem of high titre virus manufacture for gene therapy treatments. We do this with high titre, high quality, suspension transient viral manufacturing systems, and with AAV and lentiviral packaging and producer cell lines. We address the challenge of speed and consistency in gene editing with our high throughput automated CRISPR platform, and we deliver the breakthrough needed to discover antibodies to membrane proteins through novel mammalian display techniques.

MaxCyte

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.

BioNTech

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.
 

Yposkesi

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.

Thermo Fisher Scientific

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.

Plata

Merck

We are Merck KGaA, Darmstadt, Germany the vibrant science and technology company. Science is at the heart of everything we do, it drives the discoveries we make and the technologies we create. Our products and services help you safely and efficiently develop and manufacture cell and gene therapies. Our BioReliance® Services support every stage of gene therapy development – from cGMP viral vector process development and manufacturing to a comprehensive set of biosafety tests in accordance with regulatory guidelines.

In addition, our SAFC® Biopharma Raw Material Solutions is a trusted manufacturer of specialty chemicals and biologics for life sciences and high technology commercial applications. The SAFC® portfolio of custom and ready-to-use critical raw material solutions are backed by an experienced and responsive team with deep technical, quality and regulatory expertise who can tailor solutions to precisely fit your process.
 

FinVector

FinVector is a world leader in the research/development and cGMP manufacture 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. With our scientific expertise and industry knowledge FVT help clients take viral-based products from pre-clinical through to commercial phase.

Lysogene

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

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

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

Aldevron

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 additional facilities in Madison, Wisconsin and Freiburg, Germany.

VIVEbiotech

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.

STEMCELL Technologies

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.
 

Pfizer

Working together for a healthier world®
At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products. Our global portfolio includes medicines and vaccines as well as many of the world’s best-known consumer health care products.
 

Biomarin

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.

Viralgen

Viralgen Vector Core is a partnership uniquely positioned in the CDMO industry to bring forward a robust suspension platform for AAV production in a facility custom-designed to bring therapies to market as quickly as possible. Leveraging the technology platforms of AskBio, Viralgen is able to support every aspect of your AAV program from start to finish, including large-scale manufacturing, fill-finish, and quality control in a GMP-certified environment in San Sebastian, Spain. 
 

Abeona

Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene therapies for life-threatening rare genetic diseases. Abeona's lead programs include ABO-102 (AAV-SGSH), an adeno-associated virus (AAV)-based gene therapy for Sanfilippo syndrome type A (MPS IIIA) and EB- 101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB). Abeona is also developing ABO-101 (AAV-NAGLU) for Sanfilippo syndrome type B (MPS IIIB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten disease (JNCL), ABO-202 (AAV-CLN1) for treatment of infantile Batten disease (INCL), EB-201 for epidermolysis bullosa (EB), ABO-301 (AAV- FANCC) for Fanconi anemia (FA) disorder and ABO-302 using a novel CRISPR/Cas9-based gene editing approach to gene therapy for rare blood diseases. In addition, Abeona is developing its proprietary vector platform, AIM™, for next generation product candidates.

VIVET Therapeutics

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

Paragon

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

Delphi Genetics, located in Belgium, is a biotechnology company specialized in genetic engineering and plasmid DNA production.

Delphi Genetics provides innovative and flexible biomanufacturing solutions for plasmid DNA vaccines and plasmid DNA starting material for gene therapy programs. We collaborate with our customers on projects at all stages of development, from R&D to clinical phases. Our GMP-compliant single-use process for plasmid DNA manufacturing allows us to provide mg to g-scale of R&D, High Quality and cGMP-grade plasmid DNA, in our dedicated facility.

To support this manufacturing activity, Delphi Genetics also offers its proprietary STABY® "antibiotic-free" technology. STABY® technology can be implemented in all plasmid DNA and provides better plasmid stabilization and a safer way to manufacture plasmid DNA without antibiotic resistance gene, following regulatory agencies recommendations.
 

Novasep

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 20 years, Novasep has acquired experience in developing and manufacturing a wide range of viral vectors: AAV, Adenovirus, Lentivirus, HSV, VEEV, VSV…, for gene therapy, immunotherapy and vaccination.
We also offer Fill and Finish services to complete the manufacturing of your biologic.
Our recent investments in new commercial assets, addressing both drug substance and drug product manufacturing processes, will help to contribute to your project’s success. 
 

KCT

KCT is newly formed research center in Kuopio, Finland. We provide high quality basic and translational research. Professional team and laboratories with cutting edge devices are providing scientific know-how and modern technologies to develop advanced therapies. We undertake science in collaboration with academic groups all over the world. Link to FinVector's GMP manufacturing, quality and regulatory teams ensures product path from research to patients.
 

Axon Medchem



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.
 

Amicus Therapeutics

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.

Asphalion

Asphalion is an international Scientific and Regulatory Affairs consultancy, with offices in Barcelona, Madrid and Munich. We collaborate with Pharma and Biotech companies facilitating Drug Development and Regulatory Affairs projects for Drugs, Biologics, Biosimilars, ATMPs and Medical Devices. Our involvement ranges from early development, through to registration and post-commercialization phases. Since the company was founded in 2000, we have consistently grown and now have a team of over 80 employees with backgrounds in all areas of life sciences. Our consultants are experts in their fields and are in direct contact with European agencies for the implementation of new regulatory standards. We provide global services and work for hundreds of clients from around the world. Through collaborations with partners in all other continents, we can accelerate your worldwide scientific and regulatory activities by using local expertise.
Services:
• Regulatory and Scientific Strategy during Development
• Medical and Scientific Writing
• Global Submissions
• eSubmission and RIM
• Life-Cycle Outsourcing
• Pharmacovigilance
• Promoting the marketing registration of Medical Devices: Classification strategy, CE Marking and compilation of technical file. 

Sartorius

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.
 
 

Touchlight AAV

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:

  • Safer: dbDNA is produced enzymatically and minimizes bacterial sequences, thus preventing aberrant packaging of bacterial origins of replication and antibiotic resistance genes.
  • More effective: dbDNA reduces unwanted sequences. 
  • Scalable: dbDNA is manufactured from milligram to multi-gram scale with an identical process.
  • Faster to manufacture: dbDNA is manufactured in days, not months, and PO to delivery will be less than 3 months in most cases.

Research and Clinical grade dbDNA manufactured in our state-of-the-art facility in San Sebastian, Spain.
For more information, please visit: touchlightaav.com    

Clean Cells

BIOSAFETY TESTING
A full range of regulatory tests in compliance with GLP and cGMP, with more than 200 qPCR tests available. Capability to develop and validate innovative, sensitive, specific and robust tests in accordance with ICH Q2 R1.
 
GMP PRODUCTION and STORAGE
Production of Eucaryotic and Procaryotic cell banks, Viral banks and Storage in liquid nitrogen or -80°C.
 
ASSAY DEVELOPMENT
Production of cell models which express molecules of interest.
Transfection, cloning, selection, amplification.
Analysis in vitro of the cell responses: measurement of viability, proliferation, phenotype, specific functions (ex. ADCC, apoptosis, CDC), interferon activity.
Development and validation of qPCR tests: detection quantification of pathogens or contaminants, residual DNA, determination of sites of insertion of a transgene, gene copy number.
Development and validation of virus titration method. 
Detection and validation of recombinant competent replication tests (rcAAV,RCL) for gene therapy.
 
ADVANCED MEDECINE THERAPY MANUFACTURING FOR PHASE I/II
GMP manufacturing platform for bacteriophages and cell therapy products for phase I / II clinical trials.
 

Biocair

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.
 

Univercells

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.
 

AskBio

AskBio’s history-making journey began when our co-founder, Dr. Jude Samulski, discovered how the Adeno-Associated Virus (AAV) could safely deliver corrected genes to cells with genetic defects. Since then, we have been developing foundational AAV technology long before most gene therapy companies existed. Now, with the acquisition of Synpromics and the pioneering promoter and gene control advancements made by Dr. Michael Roberts, AskBio’s AAV platform is significantly enhanced.

Today, we are a multi-dimensional company with the world’s only fully integrated gene therapy platform. We have brought to life some of the industry’s most prolific AAV innovations in capsid design and high-yield AAV production with unparalleled clinical expertise and a promising therapeutic pipeline. Driven by the need to increase access and lower cost, we know the science only matters when we can see a child walk, see, hear, breathe and live a long healthy life through groundbreaking genetic medicine.

AskBio is headquartered in Research Triangle Park, N.C., U.S. with our European offices in Edinburgh, Scotland. Manufacturing is in San Sebastián, Spain through our joint venture companies Viralgen and Touchlight AAV. We welcome all who want to join us to advance gene therapy and to change the face of healthcare. Email us: careers@askbio.com.

Cevec Pharmaceuticals

CEVEC is a center of expertise for the production of biopharmaceuticals using a unique human cell-based expression system.
CAP®GT is a fully scalable manufacturing platform for viral vector production. CEVEC has successfully developed CAP®GT suspension cell-derived viral packaging cell lines, including a stable, helper virus-free AAV production platform, 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 excellent productivity for a broad range of viruses. Gene therapy vectors such as lentiviral, adenoviral, and adeno-associated viral (AAV) vectors can be produced at industrial scale.
CAP®Go enables the production of proteins “beyond antibodies”. The CAP®Go expression platform comprises a portfolio of glyco-optimized human suspension cell lines for the highly efficient production of a broad range of difficult to express recombinant proteins with authentic human post-translational modifications or on demand tailor-made glycosylation patterns.

Polyplus-transfection

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).

Cytena

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.

Ultragenyx

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.

Takara Bio

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.
 

IDTDNA

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.
 

GenIbet and iBET

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 to 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.
 

Genewiz

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.
 

Batavia Biosciences

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.
 

Greiner Bio-One International GmbH

Greiner Bio-One specialises in the development, production and distribution of high-quality plastic laboratory products and rapid test systems for adventitious agents testing.
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. Furthermore, Greiner Bio-One provides molecular analysis methods for fast and reliable detection and identification of mycoplasma and viruses in cell-culture based manufacturing processes. The entire development, manufacturing and sales operations are controlled from the German headquarter of the BioScience division in Frickenhausen.
 

PTC Therapeutics

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.
 

Cellink

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!
 

PromoCell

PromoCell is a premier manufacturer of cell culture products. We are known in particular for our broad range of human primary cells, stem cells and blood cells, optimized cell culture media, and comprehensive line of cell biology research products. Under the brand of PromoKine, we offer a wide range of products for cell analysis, apoptosis research, cell transfection, fluorescent labeling, mycoplasma detection and elimination antibodies, ELISAs, cytokines and growth factors.
 

Stilla Technologies

Stilla Technologies focuses on accelerating the development of next-generation genetic tests by providing innovative instrumentation for digital PCR. In 2016, Stilla Technologies launched the Naica System, the first and only dPCR solution to offer 3-color multiplexing. Other key features of the system are the speed, simplicity and versatility of its workflow.

Bronce

uniQure

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.  

VCN Biosciences

VCN Biosciences is a privately-owned company focused in the development of new therapeutic approaches for tumors that lack effective treatment. The company uses oncolytic adenovirus technology platform to design highly selective and efficient agents that replicate and self-amplify exclusively in tumor cells. The selectivity of VCN oncolytic adenoviruses allows their systemic administration, which is especially relevant for the treatment of disseminated cancer. Contrary to chemotherapy, the ability of oncolytic virus to self-amplify in tumor cells results in an effective dose increase with time. These properties highlight VCN candidates as promising alternatives for the treatment of refractory tumors such as pancreatic adenocarcinomas, which is the current tumor target for its most advanced candidate, VCN-01.

NLO

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, 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 Mechelen). 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.
 
 

LogicBio Therapeutics

LogicBio is a Cambridge, MA-based genome editing company focused on developing medicines to durably treat rare diseases in pediatric patients with significant unmet medical need using GeneRide, our proprietary technology platform. GeneRide enables precise, durable and site-specific integration of a therapeutic transgene without exogenous nucleases or promoters by relying on the native process of homologous recombination. Because GeneRide is designed to have this durable therapeutic effect, we are initially targeting rare liver disorders in pediatric patients where it is critical to provide treatment early in a patient’s life before irreversible disease pathology can occur. We have demonstrated proof of concept of our therapeutic platform in animal models for a number of diseases and are focusing on development of our lead product candidate, LB-001, for the treatment of Methylmalonic Acidemia, or MMA, a life-threatening disease that presents at birth.
 

Atara Biotherapeutics

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.
 

PassageBio

Passage Bio is a fully integrated genetic medicines company based in Philadelphia, PA that is developing a portfolio of six life transforming AAV-delivered therapeutics for the treatment of rare monogenic central nervous system diseases. The company was founded by industry veterans Stephen Squinto, Ph.D., and Tachi Yamada, M.D., in a partnership with gene therapy pioneer James Wilson, M.D., Ph.D.
The company has a research, collaboration and license agreement with the University of Pennsylvania and its Gene Therapy Program as well as the Penn Orphan Disease Center.