Our Partners

We are very grateful for support from our partners, please click on any logos below to head to their website.

If you would like to see your logo here, head over to our Sponsorship Guide page to find out how we can work together!

Diamond

Supersist

The goal of Supersist project is the clinical translation of new gene targeting technologies for correcting inherited mutations and empowering adoptive immunotherapy of cancer. Substantial evidence supports the therapeutic potential of ex vivo gene therapy based on Hematopoietic Stem Cell (HSC) or T lymphocytes to treat inherited diseases or cancer.

Oxford Biomedica

Oxford BioMedica (LSE:OXB) is a leading gene and cell therapy company focused on developing life changing treatments for serious diseases. The Company has built a sector leading lentiviral vector delivery platform (LentiVector®) through which it continues to develops in vivo and ex vivo gene & gene-modified therapies products both in-house and with partners. Oxford BioMedica has entered into a number of key partnerships, including with Novartis, Sanofi, GSK, Green Cross Lab Cell and Immune Design. Oxford BioMedica has world class facilities and capabilities encompassing the full range of GMP manufacturing and analytical activities to support pre-clinical, research and bioprocessing development through to GMP production and supply of clinical trial materials.  The production activities are focussed on the manufacture of lentiviral vectors from human cell lines, both in adherent and large scale serum free suspension culture. Oxford BioMedica is based across several locations in Oxfordshire, UK and employs more than 230 people.

Editas

Editas Medicine is a transformative genome editing company founded by world leaders in the fields of genome editing, protein engineering, and molecular and structural biology, with specific expertise in CRISPR/Cas9 and TALENs technologies. The company's mission is to translate its genome editing technology into a novel class of human therapeutics that enable precise and corrective molecular modification to treat the underlying cause of a broad range of diseases at the genetic level. The company has generated substantial patent filings and has access to intellectual property covering foundational genome editing technologies, as well as essential advancements and enablements that will uniquely allow the company to translate early findings into viable human therapeutic products.

Molmed

MolMed S.p.A. is a medical biotechnology company focused on research, development and clinical validation of novel anticancer therapies. MolMed’s pipeline includes antitumour therapeutics in clinical and preclinical development: Zalmoxis® (TK), a cell-based therapy; NGR-hTNF, a novel therapeutic agent for solid tumours; CAR-CD44v6, an immuno-gene therapy project; MolMed also conducts cell and gene therapy projects in collaboration with third parties, offering resources and expertise covering preclinical to Phase III trials activities. MolMed is listed on the main market (MTA) of the Milan stock exchange managed by Borsa Italiana

GSK

GSK, one of the world’s leading research-based pharmaceutical and healthcare companies – is committed to improving the quality of human life by enabling people to do more, feel better and live longer. 

AveXis

“Based in Chicago, Illinois, AveXis is a clinical-stage gene therapy company dedicated to developing and commercializing novel treatments for patients suffering from rare and life-threatening neurological genetic diseases. 
Our initial product candidate, AVXS-101, is our proprietary gene therapy product candidate currently in a Phase 1 clinical trial for the treatment of spinal muscular atrophy (SMA) Type 1, the leading genetic cause of infant mortality. SMA Type 1 is a lethal genetic disorder characterized by motor neuron loss and associated muscle deterioration, resulting in mortality or the need for permanent ventilation support before the age of two for greater than 90% of patients-
We are passionately committed to moving gene therapies into the clinical setting for patients and families devastated by rare and orphan neurological genetic diseases. With the support of industry and academic alliances, we're advancing cutting-edge science in order to treat rare and life-threatening genetic diseases—starting with our clinical-stage, proprietary gene therapy candidate, AVXS-101.”

Spark

About Spark Therapeutics Spark Therapeutics, a fully integrated gene therapy company, is seeking to transform the lives of patients with debilitating genetic diseases by developing one-time, life-altering treatments. Spark Therapeutics’ validated gene therapy platform is being applied to a range of clinical and preclinical programs addressing serious genetic diseases, including inherited retinal diseases, liver-associated diseases, such as hemophilia, and neurodegenerative diseases.  Spark Therapeutics’ validated platform has successfully delivered gene therapies with proof-of- concept data in the eye and liver. Spark Therapeutics’ most advanced product candidate, voretigene neparvovec (formerly referred to as SPK- RPE65), which has received both breakthrough therapy and orphan product designations, reported positive top-line results from a pivotal Phase 3 clinical trial for the treatment of a rare blinding condition. Spark Therapeutics’ hemophilia franchise has two lead assets: SPK-9001 in a Phase 1/2 trial for hemophilia B and SPK-8011, a preclinical candidate for hemophilia A.  

Platinum

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.

BioReliance

BioReliance was acquired by Sigma-Aldrich Corporation, in January, 2012 to supply customers access to a powerful, single-point provider whose products and services span the drug discovery, development and commercialization pipeline. As part of the SAFC business unit, BioReliance is a leading provider of cost-effective contract services, offering more than 1,000 tests or services related to biologics safety testing, specialized toxicology and animal health services. BioReliance provides outsourced services to thousands of customers from most healthcare disciplines and is the largest provider of safety testing services focused on the rapidly growing biologics sector of the pharmaceutical and biotechnology industries. BioReliance has over 700 employees and has laboratory operations in Rockville, MD and Scotland

REGENX BIOSCIENCE

REGENXBIO is a leading biotechnology company focused on the development, commercialization and licensing of recombinant adeno-associated virus (AAV) gene therapy. Our NAV® Technology Platform, a proprietary AAV gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. Our mission is to transform the lives of patients suffering from severe diseases with significant unmet medical needs by developing and commercializing in vivo gene therapy products based on our NAV Technology Platform. We seek to accomplish this mission through a combination of our internal development efforts and the efforts of our third-party licensees.

Biogen

Through cutting-edge science and medicine, Biogen discovers, develops and delivers to patients worldwide innovative therapies for the treatment of neurodegenerative diseases, hematologic conditions and autoimmune disorders. Founded in 1978, Biogen is one of the world’s oldest independent biotechnology companies and patients worldwide benefit from its leading multiple sclerosis and innovative hemophilia therapies

MeiraGTx

MeiraGTx is focused on the development of novel gene therapies for inherited and acquired disorders. The company is developing therapies for ocular diseases, including rare inherited blindness and wet and dry AMD, xerostomia following radiation treatment for head and neck cancer, and neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS). MeiraGTx also has an innovative gene regulation platform that provides the potential to expand the way gene therapy can be applied to create a new paradigm for biologic therapeutics.

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 gene therapy company committed to discovering and developing novel medicines that can offer life-changing benefits to patients living with rare diseases or diseases of the eye who currently have limited or burdensome treatment options. Adverum has a robust pipeline and is leveraging its next-generation adeno-associated virus (AAV)-based directed evolution platform to generate product candidates designed to provide durable efficacy by inducing sustained expression of a therapeutic protein. Our focus on the patient is supported by clinical development expertise and core capabilities in vector optimization, process development, manufacturing, and assay development. 

Gold

Genethon

Genethon, created by 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 muscular dystrophies, immune deficiencies, blood, ocular and liver diseases. These products are developed either with Genethon as sponsor, or in partnership with private companies and academic institutions.

Sangamo

Sangamo BioSciences (Nasdaq: SGMO) Sangamo BioSciences, Inc. is focused on the development of genetic therapies based on its zinc finger protein (ZFP) technology platform for genome editing and gene regulation, and its AAV-cDNA gene therapy platform. In 2016, the Company expects to initiate a Phase 1/2 clinical trial for its zinc finger nuclease (ZFN)-based therapeutic for the treatment of hemophilia B, which represents the first in vivo genome editing application in man. Sangamo also plans to file an Investigational New Drug (IND) application to initiate a Phase 1/2 clinical trial for hemophilia A based on its AAV-cDNA gene therapy approach. In addition, the Company is developing ZFN-based therapeutics for lysosomal storage disorders, including MPS I (Hurler syndrome) and MPS II (Hunter syndrome), and has strategic collaborations with Biogen Inc. to develop therapeutics for sickle cell disease and beta-thalassemia, and with Shire International GmbH for Huntington’s disease. For more information about Sangamo, visit the Company’s website

Dimension Therapeutics

Dimension Therapeutics is a gene therapy company focused on developing novel treatments for rare diseases. The company was launched in 2013 by Fidelity Biosciences and REGENX Biosciences. Dimension has now raised $30M with funding from Fidelity Biosciences and OrbiMed. Our team comprises biotech industry veterans and thought leaders in gene therapy and rare diseases.

We are focused on advancing our platform of gene therapy programs in rare diseases through clinical development, starting with lead programs in hemophilia, and building out a world-class product engine for adeno-associated virus (AAV) therapeutics across a wide array of single-gene rare diseases.

Finnvectors

FinVector is a world leader in the research and development of Viral-Based Gene Therapy products, with state-of-the-art facilities and a highly experienced scientific team working in the gene therapy market. We deliver a tailored service to meet and exceed our clients’ needs, and use our scientific expertise and industry knowledge to help clients take viral-based products from the pre-clinical phase, through clinical trials and to the market.

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.

Bayer

Bayer: Science For A Better Life

Bayer is a global enterprise with core competencies in the Life Science fields of health care and agriculture. Its products and services are designed to benefit people and improve their quality of life. 

About Hematology at Bayer

Bayer is committed to delivering science for a better life by advancing a portfolio of treatments. Hematology at Bayer includes numerous compounds in various stages of development for hemophilia, sickle cell anemia, and other blood and bleeding disorders. Together, these compounds reflect the company’s commitment to research and development, prioritizing specific targets for intervention with the potential to improve the way that rare blood and bleeding disorders are treated.

Oncorus

Oncorus, Inc. is an early-stage biotechnology company developing a next-generation immunotherapy platform to treat cancer. Oncorus’s technology platform, based on innovative advancements with oncolytic viruses, has the potential to treat many tumor types, including highly malignant and aggressive cancers. Oncorus was founded by leading academic scientists and biotechnology entrepreneurs, including Mitchell H. Finer, PhD, an industry veteran and Managing Director of MPM Capital. A leader in corporate philanthropy, Oncorus has taken a pledge to donate a portion of product sales to fund promising cancer research and to support cancer care in the developing world. Oncorus is headquartered in Kendall Square, Cambridge, Massachusetts.

CellforCure

A company of the LFB group, CELLforCURE is a key industrial CDMO in the field of advanced therapy medicinal products.

CELLforCURE is a pharmaceutical company, and has been a pharmaceutical establishment since 2013, acting as a CDMO (Contract Development and Manufacturing Organization) at a large scale, in compliance with GMP and cGMP requirements.

CELLforCURE proposes a one stop shop services from bench to patient and market, including:

  • Optimization and industrialization of processes
  • GMP manufacturing of clinical and commercial batches of cell and gene therapy product
  • Regulatory services and pharmaceutical distribution

CELLforCURE’s facility, the largest European facility for GMP manufacturing of cell and gene therapy products is located in Les Ulis, near Paris (France).

CELLforCURE’s facility design is very flexible allowing to meet clients needs for large scale manufacturing of all types of autologous and allogeneic cell and gene therapy products, using open, closed, automated “best in class” technologies.

CELLforCURE optimizes and improves your production process for a better robustness, scalability, cost efficiency and pharmaceutical compliance.
All operations are conducted in GMP and cGMP compliance.

CELLforCURE has an important experience, in the processing of a broad spectrum of cell types for autologous, and allogeneic indications.

CELLforCURE has a strong team with a real knowledge and experience in cell and gene manufacturing.

Silver

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

AFM

AFM (French Muscular Dystrophy Association) has a single objective: to defeat neuromuscular diseases, which are devastating muscle-wasting diseases. Created in 1958 by a group of patients and their families, and recognised as being of public utility in 1976, it has set itself two missions: curing neuromuscular diseases and reducing the disabilities they cause.

www.afm-france.org

Genosafe

GenoSafe® is a full contract research and consulting organization which specializes in evaluating the quality, safety and efficacy of innovative biological products.

We meet our clients’ specific needs by performing custom studies in strict compliance with regulatory requirements.

GenoSafe is a Good Laboratory Practices (GLP) compliant facility .

GenoSafe is eligible for the CIR (research tax credit). Our clients may include the R&D Services performed at GenoSafe in their own research tax credit calculation.

Eufets

EUFETS (Germany) is a Contract Development and Manufacturing Organisation specialised in the industrialisation of cell and gene therapy products (viral vectors, cell products and in vitro transcribed mRNA). Based on extensive expertise in molecular biology, virology and cell biology as well as an understanding of the regulatory prerequisites, our GMP experts support you to develop and manufacture your products in a safe and cost-efficient way. We offer a complete service spectrum from process and assay development through clinical trial medication to in-market supply in our state-of-the-art GMP facility.

Come and visit us at booth 14.

Nature

The official journal of the American Society of Gene and Cell therapy, Molecular Therapy is dedicated to publishing important peer-reviewed research and cutting-edge reviews and promoting the sciences in genetics, medicine and biotechnology, and is the parent journal to the open access titles Molecular Therapy – Nucleic AcidsMolecular Therapy – Methods & Clinical Development and Molecular Therapy – Oncolytics.

TargetAMD

Age-Related Macular Degeneration (AMD) is a rapidly progressing, blinding disease that appears to result from age-associated alterations that include cell degeneration and vessel grow through Bruch’s membrane into the subretinal space. Today’s treatment includes repeated, frequent injections of VEGF (Vascular Endothelial Growth Factor) antibodies. PEDF (Pigment Epithelium-Derived Factor) as physiological antagonist of VEGF should also inhibit the pro-angiogenic acting VEGF. The overall objective of TargetAMD is to deliver PEDF by using the hyperactive Sleeping Beauty (SB100X) transposon system in a cell-based, non-viral gene therapy in two clinical phase Ib/IIa trials.

aldevron

Aldevron is a recognized leader in contract manufacturing and development services for nucleic acids, proteins and antibodies. Founded in 1998, we provide companies with essential components for research, clinical and commercial applications. Our products have supported numerous programs in gene therapy, cell therapy and regenerative medicine from the bench to the bedside. Aldevron’s services include GMP-Source™ and GMP plasmid manufacturing, linear DNA and mRNA production, gene synthesis, RNA synthesis enzymes and fully human and recombinant antibody generation. Our collaborative approach and commitment to providing quality materials allow us to meet precise client requirements and provide innovative solutions to advance science. Aldevron’s headquarters is in Fargo, North Dakota and has facilities in Madison, Wisconsin and Freiburg, Germany.

Cobra Bio

About Cobra Biologics: Cobra Biologics is a leading international clinical and commercial manufacturer of biologics and pharmaceuticals with three GMP approved facilities. We offer a broad range of integrated and stand-alone development services, stretching from cell line development through to the commercial supply of investigational medicinal product. We take pride in manufacturing excellence and being a trusted provider, delivering what we promise and helping our customers to develop drugs for the benefit of patients. Cobra Biologics provides manufacturing solutions to the biologics and pharmaceutical industry covering antibodies, recombinant proteins, viruses, DNA, cellular therapeutics and small molecules.

Oxford Genetics

Oxford Genetics is a synthetic biology company taking an engineering approach to DNA, virus and cell line design, optimization and development. The business has a number of technologies enabling the delivery of complex solutions for the life sciences, including proprietary plasmids for protein manufacture, and improved lentivirus production systems for gene therapy applications. These systems include proprietary promoters to maximize yield and have been developed using proprietary bioinformatics software for gene optimization. Our proprietary bioinformatics and DNA design algorithms are designed to improve recombinant protein expression. Oxford Genetics Ltd has a track record of delivering DNA design and cell line development projects for the improved production of antibodies, vaccines and viruses.

Stemcells 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.
 

Orchard therapeutics

Orchard Therapeutics is a clinical-stage biotechnology company with operations in London and the United States and dedicated to bringing transformative gene therapies to patients with serious and life-threatening orphan diseases. 

Pfizer

Pfizer and Rare Diseases
Rare diseases are among the most serious of all illnesses and impact millions of patients worldwide, representing an opportunity to apply our knowledge and expertise to help make a significant impact in addressing unmet medical needs. The Pfizer focus on rare diseases builds on more than two decades of experience, a dedicated research unit focusing on rare diseases, and a global portfolio of more than 20 medicines approved worldwide that treat rare diseases in the areas of hematology, neuroscience, inherited metabolic disorders, pulmonology, and oncology.

Pfizer Inc: 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. 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, Pfizer has worked to make a difference for all who rely on us. For more information, in addition, to learn more, follow us on Twitter at @Pfizer and @Pfizer_News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

Brammer Bio

Brammer Bio is a contract development and manufacturing organization (CDMO) serving companies seeking to develop and commercialize cell and gene therapies. With an experienced management team and exceptional scientific expertise and proven manufacturing capabilities we offer the knowledge and resources necessary to help you deliver innovative cell and gene-based therapies.

Chiesi

Headquartered in Parma, Italy Chiesi Farmaceutici is an international research-focused Healthcare group, with over 80 years of experience in the pharmaceutical industry. Chiesi researches, develops and markets innovative drugs in the respiratory therapeutics, specialist medicine and rare diseases areas. Its R&D centres in Parma (Italy), Paris (France), Cary (USA), Chippenham (UK) and the R&D team of the acquired Danish company Zymenex, integrate their efforts to advance Chiesi's pre-clinical, clinical and registration programs. Chiesi employs over 4,500 people, 560 of whom are solely dedicated to Research and Development activities.

Bronze

TiGenix

TiGenix is a leading European cell therapy company with a commercial product and a strong clinical stage pipeline of adult stem cell programs. The company?s lead product, ChondroCelect®, for cartilage repair in the knee, is the only approved cell-based product in Europe, and is currently being launched across Europe. TiGenix?s adipose derived allogeneic stem cell platform has been extensively validated. TiGenix is based out of Leuven (Belgium) and has operations in Madrid (Spain), and Sittard-Geleen (the Netherlands).

Genzyme

Genzyme has pioneered the development and delivery of transformative therapies for over 30 years. Founded in 1981 in Boston, Massachusetts, Genzyme evolved from a tiny start-up with just a handful of employees to one of the world’s leading biotech companies. Acquired by Sanofi in 2011, Genzyme now benefits from the reach and resources of one of the world’s largest pharmaceutical companies, with a shared commitment to improving the lives of patients.

Genzyme has long been known for our expertise in the class of rare genetic diseases known as lysosomal storage disorders (LSDs). LSDs remain the heart of our company today, but we have also expanded – through both in-house development and strategic acquisitions and partnerships – to other disease areas such as thyroid cancer and multiple sclerosis.

Apceth

apceth - The cell engineering company!

PROPRIETARY TECHNOLOGY & PRODUCTS

• Clinical stage biopharmaceutical company

• Leader in the development of genetically engineered cell therapies

• Native and engineered (2nd generation) mesenchymal stem cells

• Cancer, immunomodulation and tissue regeneration

CONTRACT DEVELOPMENT & MANUFACTURING ORGANIZATION

• Reliable and high-performance partner

• Complex cell-based and gene therapy products (ATMPs)

• Product and process development, GMP manufacturing

• GMP-certified since 2010

Genenta

Genenta Science develops a gene transfer strategy into autologous hematopoietic stem cells (HSCs) to target interferon-α expression to tumor-infiltrating monocytes/macrophages. An HIV-derived and genetically disabled viral vector - Lentivirus - delivers the gene into the HSCs. Type I Interferons have been shown to promote tumor immunity, but systemic toxicity has limited their use. The innovative therapy of Genenta Science, by combining transcriptional and microRNA-mediated control, enables tumor-infiltrating monocytes/macrophages to selectively express interferon-α limited to the tumor area, thus reducing its toxicity

Supporter

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