Platinum Sponsor

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.

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

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.

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.

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.

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 or AAV 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.

Bronze Sponsor

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.

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.

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.