Diamond Sponsor

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.

MaxCyte

MaxCyte is a leading provider of cell-engineering platform technologies that are driving the next-generation of cell-based therapies and making a meaningful difference for patients. The Company's technology is employed by leading drug developers worldwide, including all of the top ten global biopharmaceutical companies. MaxCyte has granted 13 strategic platform licenses to leading cell-based therapy developers. Through 2020, MaxCyte has granted licenses for more than 140 cell therapy programs, with over 100 licensed for clinical use. Our Flow Electroporation® technology and next-generation ExPERT® platform enable our partners to accelerate, streamline, and improve the drug development process from the early stages of research to commercialization. MaxCyte was founded in 1998 and is headquartered in Gaithersburg, Maryland, U.S.

PTC Therapeutics

PTC is a science-driven, 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 and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. To learn more about PTC, please visit us at www.ptcbio.com and follow us on Facebook, on Twitter at @PTCBio, and on LinkedIn.

Platinum Sponsor

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

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.

REGENXBIO Inc.

REGENXBIO Inc. is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. Our gene therapy product candidates are designed to deliver functional genes to address genetic defects in cells, enabling the production of therapeutic proteins or antibodies that are intended to impact disease. Through a single administration, gene therapy could potentially alter the course of disease significantly and deliver improved patient outcomes with long-lasting effects.  We have developed a broad pipeline of gene therapy programs using our NAV® Technology Platform to address genetic diseases through two modalities: AAV-mediated antibody delivery and monogenic gene replacement. We believe this platform forms a strong foundation for our current programs and with our ongoing research and development, we expect to continue to expand the platform.

Sarepta Therapeutics

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, totalling 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 fuelled 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.

Gold Sponsor

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

Our mission: Delivering innovative and trusted scientific solutions across the globe

Cevec Pharmaceuticals

CEVEC is a leading provider of high-performance cell technology for the manufacturing of advanced bio-therapeutics from R&D to manufacturing scale. The company’s product portfolio comprises platform technologies for gene therapy viral vectors, vaccines and complex recombinant proteins. With the ELEVECTA® Technology, CEVEC offers a unique solution for large-scale production of AAV vectors using helper virus-free producer cell lines with all necessary components stably integrated into the cell. The technology is based on suspension cells and does not require any expensive transfection reagents and cGMP plasmids. CEVEC’s CAP® Technology based on human suspension cells is the ideal production platform for RCA-free Adenoviral vectors, Lentiviral vectors, viral vaccines and exosomes. With the CAP-Go® Technology CEVEC provides a solution to the increasing need for recombinant production of complex and highly glycosylated protein molecules, including laminins, coagulation factors and plasma proteins.

Genethon

Genethon, created by the AFM-Telethon, has the mission to make innovative  gene therapy treatments available to patients affected with rare genetic diseases. To meet this challenge Genethon has assembled the technical and human resources needed to accelerate the medical application of scientific discoveries arising from fundamental research. Strong translational research programs engage multi-disciplinary teams and are supported by a first rate technological platform and cGMP facility

The pipeline of Genethon includes products currently in international clinical trials and at preclinical stages, for immune deficiencies, muscular dystrophies, ocular and liver diseases. These products are developed either with Genethon as sponsor, or in partnership with private companies and academic institutions.

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.

uniQure

uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with CNS, liver/metabolic and cardiovascular diseases

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).

 

Silver Sponsor

Adverum Biotechnologies

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.

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.

GenoSafe

GenoSafe provides analytical testing services for the evaluation of quality, safety and efficacy of Gene and Cell therapy candidates including:

· GLP biodistribution, shedding and immunogenicity studies ;

· QC testing, such as viral titration, safety and potency/efficacy testing ;

· GCP bioanalysis of patient samples, including immunogenicity, shedding and gene expression studies.

GenoSafe brings more than 15 years of experience in the development, qualification and validation of key analytical methods for product and sample testing.

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.

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

PlasmidFactory

PlasmidFactory GmbH & Co. KG is a globally active biopharmaceutical company, founded in Bielefeld, Germany, in 2000. The leading contract manufacturing organization (CMO) for plasmid and minicircle DNA 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 client's requirements in modern laboratories with high quality standards.

The company's R&D activities are focused on its core competencies: Production, analysis and storage of plasmid and minicircle DNA. Furthermore, PlasmidFactory co-operates with national and international partners on individual projects, e.g. in the fields of CAR-T cell, AAV production and mRNA production.

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.

VGXI Inc

VGXI, Inc. is a leading contract manufacturer of DNA-based pharmaceuticals with 20 years of experience providing high quality cGMP products to clinical trials worldwide. Uses include DNA vaccines, immunotherapies, and cell and gene therapy applications. The company's continuous, low-shear AIRMIX® lysis technology and optimized purification process provide highly supercoiled plasmid with exceptional purity across all manufacturing scales. Production services include high quality plasmid for preclinical research, Highly Documented (HD) plasmid as a critical raw material for GMP viral vector production or pharm/tox studies, and cGMP plasmid DNA for clinical through commercial supply. Pre-Clinical through GMP RNA manufacturing services are also available.

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.

Bronze Sponsor

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.

Greiner Bio-One International GmbH

Greiner Bio-One is a leading partner for the diagnostic and pharmaceutical industry as well as biotechnology with laboratory products made of plastic.
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 headquarter of the BioScience division in Frickenhausen.
For detailed information to our products and solutions please refer to our homepage or get in touch with our product specialist:
Dr. Mareike Langbein
Product Manager
M: +49 152 567 71 392

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.

Sirion Biotech

SIRION Biotech GmbH is Europe’s leading commercial supplier of viral vector technologies (AAV, LV, AV). Our viral vector know-how enables engineering for a new generation of optimized vectors, in R&D grade as well as clinically-compliant.

We guide our customer through the entire Cell & Gene Therapy value chain – from capsid evolution and clinical vector design to reduce vector dose and improve therapeutic success, to supporting them with our process and development experience and manufacture of high-quality viral vectors for late preclinical applications.

We also offer LentiBOOST®, a highly effective lentiviral transduction enhancer for therapeutic cell types like CAR-T cells and CD34+ hematopoietic stem cells. LentiBOOST® is currently included in more than 20 Phase III and I/II clinical trials.

The company is easily accessible with headquarter in Munich, as well as offices and agents in Boston, Paris, Tokyo, Seoul and Tel Aviv. Commercial arrangements range from fee-for-service and IP-generating collaborations to milestone & licensing arrangements.

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 mostcommonly 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.