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Platine

AVROBIO

AVROBIO’s mission is to free people from a lifetime of genetic disease with a single dose of gene therapy. We aim to halt or reverse disease throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a program in Pompe disease. AVROBIO is powered by the plato™ gene therapy platform, our foundation designed to scale gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit our website, and follow us on Twitter and LinkedIn.

CSL Behring

Driven by Our Promise
It began with a promise. CSL Behring's parent company, CSL, was formed more than 100 years ago to save lives using the latest technologies. In the century since, CSL Behring has grown into a global biotechnology leader, driven by that same promise to save and improve lives. We offer the broadest range of quality plasma-derived and recombinant therapies in our industry. Our ability to innovate and deliver life-saving medicines for patients with rare disease and other unmet medical needs around the world has earned us a reputation for always putting patients first. We are CSL Behring and we are driven by our promise.
 

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

Pfizer France

Pfizer Inc.: Breakthroughs that change patients’ lives®

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, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, we have worked to make a difference for all who rely on us.

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.

Thermo Fisher Scientific

As the world leader in serving science, Thermo Fisher Scientific is uniquely positioned to provide the quality materials, services and support need to accelerate the pace of advanced therapy development. We understand the complexity of this rapidly-evolving industry and have made significant investments in cGMP raw material manufacturing and drug product manufacturing capabilities to provide innovative end-to-end solutions- from the acquisition of Brammer Bio to investing to expand our critical raw material manufacturing facilities. Partner with us to access the high-quality materials, services, and support you need from discovery to clinical research and commercial cell and gene manufacturing. Through our Thermo Scientific, Applied Biosystems, Invitrogen, Fisher Scientific, Unity Lab services, Patheon, and Gibco brands, we offer an unmatched combination of innovative technologies, manufacturing, and distribution capabilities.

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.

Or

Beckman Coulter Life Sciences

Beckman Coulter Life Sciences is dedicated to developing and providing advanced technologies and equipment for research and discovery to explore new treatment methods. Our products include Liquid Handling and Genomic solutions, Particle Counting and Characterization, Centrifugation and Flow Cytometry, which are implemented in all major areas of Life Sciences such as biology, biochemistry, biophysics, nanotechnology and molecular biology to simplify and automate existing processes in the lab.

Our vision: Advancing science through discovery

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.

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.
 

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 marked three impressive decades by unveiling a dramatic new redesign and a renewed editorial mission. Human Gene Therapy serves as the Official Journal of ESGCT.

Yposkesi

YPOSKESI, based in Corbeil-Essonnes, near Paris (France), is one of the largest European Contract Development and Manufacturing Organizations dedicated to the development and production of gene and cell therapy treatments. The broad expertise of YPOSKESI covers the production of Lentivirus & AAV viral vectors based on transfection processes, for pre-clinical development and clinical trials in compliance with the standards of Good Manufacturing Practice regulations. YPOSKESI benefits from the 30 years viral vector expertise of its long-standing partner, GENETHON, a major player in gene therapies for rare disease.
 
In addition, YPOSKESI has an experienced and efficient team of nearly 190 experts in Bioproduction, Quality Assurance, Quality Control, Project Management and Process Development, operating in a 5,000m2 (approx. 53,819 sq ft) modern GMP facility. YPOSKESI is also significantly investing in the development of innovative technologies for large-scale cost-effective manufacturing of these highly complex Advanced Therapeutics Medicinal Products. YPOSKESI plans to extend its production capacity by 2022, with an additional building including two suites dedicated to commercial manufacturing activities with a total of 10,000 m² (approx. 107639 sq ft) space.
 
By forging a solid industrial partnership with the South-Korean SK group, its majority equity shareholder, YPOSKESI reinforced its industrial strategy based on the technological expertise of SK pharmteco, the US SK’s affiliate, complementing that of YPOSKESI, and its solid experience in international manufacturing as a contract manufacturing organisation (CMO).
 

Argent

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.

ArcticZymes Technologies

ArcticZymes Technologies is a Norwegian biotechnology company which has specialized in unique cold adapted marine enzymes for gene technology applications. These enzymes may simplify workflows due to their unique properties such as salt-tolerance, heat-lability and high activity at low temperatures. All our enzymes are produced recombinantly at very high purity.

ArcticZymes offer unique enzymes for various market segments like manufacturers of molecular diagnostic kit, pharmaceutical bioprocessing, academic research, clinical laboratories and manufacturers of reagents for the Life Science industry.

e-Zyvec

e-Zyvec is a startup offering services in molecular biology to research laboratories, CROs and Biotech industries that are using genetic engineering approaches.
Our offer is to produce tailor-made ready-to-use gene expression vectors (plasmids) and the expertise to design such molecules so they will really fit our customers’ needs. We are exclusively exploiting a patented DNA assembly method that enable us to modularly assemble vectors from up to 12 DNA fragments in an ordered and seamless way in a single-tube single-step reaction. Our process has been optimized and streamlined so we can carry out multiple assemblies simultaneously.
Building vectors with us is unrestrictive, easy, fast and accurate.
 

Eurogentec

The Bio-Manufacturing business unit is a cGMP accredited Contract Manufacturing Organization (CMO) that produces recombinant proteins and plasmids from microbial fermentation and IVT-RNA for clinical and commercial phases.
Eurogentec provides a wide range of services: process transfer and development, cGMP manufacturing, Quality Control, bulk release.
 
In 2020, Eurogentec launched a 1500L commercial line.
 
Plasmid: Cost-effective manufacturing of GMP starting material or injectable API pDNA.
rProtein: Significant experience in the protein production from Yeast (P.pastoris, S.cerevisiae, etc) and bacteria (E.coli).
IVT-RNA: Eurogentec is a pioneer in GMP in vitro transcript RNA. Our new facility can adapt to many different processes including availability of various modifications (co- or post-transcription).
 
With over 20 years of experience as CMO, you benefit from our broad manufacturing experience. Our philosophy is to establish a close partnership with our clients, adapt to their project needs and to provide them with cost and time efficient production processed.
 

Miltenyi Biotec

Miltenyi Biotec is a global provider of products and services that advance biomedical research and
cellular therapy. Since 1989, we have developed innovative and reliable technologies for scientists
and clinicians around the world. Our integrated portfolio of tools covers techniques of sample
preparation, cell separation, flow cytometry, cell culture, molecular analysis, and preclinical imaging.
The MACS® brand has set standards worldwide and is trusted across basic, translational, and clinical
research settings. Our expertise covers research areas like immunology, stem cell biology,
neuroscience, and cancer, and clinical research areas that include hematology, graft engineering, as
well as apheresis. We are committed to supporting scientists and the scientific community – from
comprehensive technical support to training at our MACS Academy. Today, we are more than 2,000
employees in 22 countries – all dedicated to providing solutions that empower scientific discovery
and advance cellular therapy.
 

Myriade

Myriade is a French company created in 2017 that develops an innovative nanoscale imaging technology: the VIDEODROP. Based on the principles of interferometry, the Videodrop makes it possible to visualize living nanoparticles in the range of 30 nm and 10 microns without labeling, in real time and in a single drop. The Videodrop processing algorithms compute the concentration and size distribution of the nanoparticles and enable to analyze complex mixtures of phages (T4, lambda) for applications in phagotherapy, continuously monitor viral vector solutions (Lentiviruses, adenoviruses) for gene and cell therapy, and distinguish the different types of EVs to vectorize, diagnose or treat.
 
 

OriGen Biomedical

OriGen Biomedical manufactures a complete line of products for cryopreservation and cell culture.
OriGen products are CE-marked and ISO 13485:2016 compliant. OriGen produces closed-system cell culture single use solutions including the PermaLife Cell Culture Bag. The industry-preferred CryoStore Freezing Bag is designed for cryopreservation of stem cells in ultra-low temperatures. An Overwrap is available which has excellent low temperature freezing properties to provide additional protection for your primary product. All of OriGen’s products are available in a variety of sizes and configurations to meet your lab’s needs.

Polyplus-transfection

Polyplus-transfection applies its 20 years expertise to the development of novel transfection solutions. Polyplus-transfection is the leading supplier of a key critical component for viral vectors manufacturing for Gene & Cell Therapy. We are proud to provide qualified and GMP-grade transfection reagents suitable from R&D to clinical trial and commercial scale, accompanied by a strong scientific and regulatory support. In addition, we provide a range of effective transfection reagents to deliver most nucleic acids, including DNA and siRNA in vitro and in vivo.
 

Progen

PROGEN, founded in 1983 and located in Heidelberg, Germany is an established manufacturer and supplier of AAV Titration ELISA tests and antibodies for gene therapy research and development.  In the past 20 years, the DIN EN ISO 13485 certified company has established a unique portfolio of AAV tools for basic and clinical research as well as for pharmaceutical applications. Among them are specific antibodies to study AAV assembly, capsid formation or the course of an infection as well as a line of reliable AAV quantification ELISAs for different serotypes utilizing PROGEN’s portfolio of capsid-specific AAV antibodies. PROGEN offers its broad expertise in antibody & ELISA technologies, protein interaction and purification and welcomes collaborations with academic and industrial institutions to advance basic AAV research. In addition, the company aims to establish partnerships with academic, pharmaceutical or medical institutions for the development of AAV-based tools and therapies, e.g. for standardization and validation steps in clinical trials.
 

Bronze

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.

Forge Biologics

Forge Biologics is a hybrid gene therapy contract manufacturing and therapeutics development company.  Forge’s mission is to enable access to life changing gene therapies and help bring them from idea into reality.  Forge has a 175,000 ft2 facility in Columbus, Ohio, “The Hearth,” to serve as its headquarters.  The Hearth is the home of a custom-designed cGMP facility dedicated to AAV viral vector manufacturing and will host end-to-end manufacturing services to accelerate gene therapy programs from preclinical through clinical and commercial stage manufacturing.  By taking a patients-first approach, Forge aims to accelerate the timelines of these transformative medicines for those who need them the most.
 

Freeline

Freeline is a clinical-stage, fully integrated, next generation, systemic AAV-based gene therapy company with the ambition of transforming the lives of patients suffering from inherited systemic debilitating diseases.
A life-changing breakthrough
Our ambitious vision is to create better lives for people suffering from inherited, systemic debilitating disease.
We aim to deliver one-time gene therapy treatments that provide functional cures through permanently sustained physiological protein levels, leveraging the high expression enabled by our proprietary gene therapy platform. Our initial focus is on developing treatments for monogenic diseases with high unmet need followed by expansion to address diseases requiring higher protein expression.
Our next generation gene therapy uses AAV technology to deliver safe and effective gene replacement to the liver to produce sustained therapeutic protein expression for diseases like haemophilia B and Fabry disease. Our treatment builds on the pioneering work of our founder, Amit Nathwani, Professor of Haematology at UCL. His award-winning scientific research was the first to show successful and sustained correction of bleeding symptoms in patients with severe haemophilia B.
Transferrable technology
We plan to use our novel proprietary capsid across our entire pipeline, leveraging faster and cost-effective pathways to clinic for our different indications. We are working hard on our vision to build and advance a pipeline of curative treatments to make a life-changing impact.

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.

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.

Plasmid Factory

PlasmidFactory GmbH & Co. KG is a globally active biopharmaceutical company, founded in Bielefeld, Germany, in 2000. As a leading contract manufacturing organization (CMO) for plasmid and minicircle DNA, it has a strong customer base in the fields of cancer research, gene and cell therapy, CAR-T-cell development, and vaccination. PlasmidFactory produces plasmids and minicircles according to customer's requirements in modern laboratories with high quality standards. In addition to its In Stock products, e.g. for the worldwide exclusively offered pDG/pDP AAV 2-plasmid-system, and custom manufacturing of plasmid and minicircle DNA, PlasmidFactory focuses its R&D efforts on its core competencies in the production, analysis, application, and storage of DNA.

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.
 

Xell AG

Xell is an innovative partner for the biotech and pharmaceutical industry, providing efficient solutions in cell culture technology. Based on its proprietary technology, Xell has developed a range of customized as well as commercial culture media and feeds for the most commonly used mammalian cell types (CHO, HEK, BHK, MDCK, Hybridoma etc). Our chemically defined products are animal-component free and compliant for GMP manufacturing. Apart from the media platform we offer fast and reliable analytical services - among others, proprietary methods for amino acid, vitamin, (trace) element, organic acid or polyamine analyses. Additionally, cell culture and process services are provided to better understand cellular behavior and optimize cultivation processes. At our production site, we offer large scale production of liquid and powder media as well as related solutions approved by ISO 9001 QMS.