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.
 

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.
 

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 3,800 employees in 28 countries – all dedicated to providing solutions that empower scientific discovery and advance cellular therapy.

Myriade

Myriade develops the VIDEODROP, an innovative nanoscale imaging technology. 

Based on the principles of interferometry, the Videodrop makes it possible to measure the size and concentration of biological nanoparticles like lentivirus, adenovirus or retrovirus 

• in real time (40s) 
• in a single drop (5µL) 
• between 80nm & 500 nm 
• in a concentration range of 10E8 to 10E10 part/ml 
• without labelling & no purification 
• on viscous & complex samples 

• Continuously monitor bioproduction processes thanks to its rapidity of measurement (40s) 
• Work on real samples at any stage of the bioproduction in a non-denaturant way 
• Control the yield of vector production after harvesting, Purification, or Concentration steps 
• Quickly identify lot-to-lot variations 

Providing linear results compared to p24 ELISA, VIDEODROP is suitable for "at-line" characterization of lentiviral vectors and in-process controls. 

 

Pall Corporation

Pall corporation offers fully integrated end-to-end bioprocessing solutions for gene therapy, from early process development projects to commercialization. Our dedicated teams of experts will work closely with you to overcome development and viral vector manufacturing obstacles to accelerate delivery of commercially viable gene therapies to patients.
 
Our extensive portfolio of upstream, downstream, and formulation and filling technologies is suitable for manufacturing different viral vector types in adherent or suspension-based processes.
 
Whether that means quickly developing a process, helping you ensure regulatory compliance, or making existing processes easier, faster and better, we are ready to work with you to address your process needs and accelerate your speed to market.
 
Please visit www.pall.com/en/biotech/gene-therapy
 

PeproTech

PeproTech, part of Thermo Fisher Scientific, specialises in the development and manufacture of high-quality cytokine products for the life-science and cell therapy market. 

With more than 30 years’ manufacturing experience, PeproTech’s range of RUO, Animal-Free and GMP cytokines offers the performance and reliability that you need.

With over 2,000 products, PeproTech has developed and refined innovative protocols to ensure quality, reliability and consistency.

Our mission is to provide the highest quality products and premium support that address the needs and demands of today's scientists and researchers.

We pride ourselves on being a trusted partner within the scientific community.

Please contact PeproTech to request a quote or discuss your research requirements:

Tel: 020 7610 3062 or Email: info@peprotech.co.uk

Polyplus-transfection

Polyplus is a leading upstream solutions provider for advanced biologic and cell and gene therapy production from research to commercial scale. An innovator in nucleic acid delivery, the legacy portfolio features process-centric transfection reagents, kits, and support services. Custom plasmid vector design was integrated into the offer in 2022 as a first measure to expanding the products and services portfolio to help customers (or the industry) optimize process economics while meeting strict scientific and regulatory standards. Headquartered in Europe, the Polyplus team continues to grow globally with operations in the United States and Asia.

SCIEX

SCIEX helps to improve the world we live in by enabling scientists and laboratory analysts to find answers to the complex analytical challenges they face. The company's global leadership and world-class service and support in the capillary electrophoresis and liquid chromatography-mass spectrometry industry have made it a trusted partner to thousands of the scientists and lab analysts worldwide who are focused on basic research, drug discovery and development, food and environmental testing, forensics and clinical research.
 
With over 40 years of proven innovation, SCIEX excels by listening to and understanding the ever-evolving needs of its customers to develop reliable, sensitive and intuitive solutions that continue to redefine what is achievable in routine and complex analysis. 
 

STEMCELL Technologies

At STEMCELL, science is our foundation. Driven by our mission to advance research globally, we offer over 2,500 tools and services supporting discoveries in stem cell research, regenerative medicine, immunotherapy, and disease research. By providing access to innovative technologies such as organoids, gene editing, and specialized media, we’re helping scientists accelerate the pace of discovery.
 

VectorBuilder

As a global pioneer in custom DNA vectors and recombinant viruses, VectorBuilder’s revolutionary online-to-offline (O2O) platform provides a powerful one-stop solution to all the vector and virus needs in the life sciences. VectorBuilder has a wide spectrum of offerings, including:

- Vector design
- Custom cloning
- Virus production (AAV, adenovirus, lentivirus, etc.)
- AAV capsid evolution and biodistribution services
- mRNA production
- Library construction
- Recombinant Protein Production
- Stable cell line generation
 
Additionally, our state-of-the-art GMP facilities provide GMP-grade plasmid and virus manufacturing for a wide range of clinical applications. 
 
 

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.

Cytiva

Cytiva is a global life sciences leader dedicated to advancing and accelerating therapeutics. Cytiva is a trusted partner to customers that undertake life-saving activities ranging from biological research to developing innovative vaccines, biologic drugs, and novel cell and gene therapies. Cytiva brings speed, efficiency and capacity to research and manufacturing workflows, enabling the development, manufacture and delivery of transformative medicines to patients.

Gilead

Gilead Sciences, Inc. is a research-based biopharmaceutical company that discovers, develops and commercializes innovative medicines in areas of unmet medical need.  The company strives to transform and simplify care for people with life-threatening illnesses around the world. Gilead has operations in more than 35 countries worldwide, with headquarters in Foster City, California, USA.
Kite, a Gilead Company, is a biopharmaceutical company based in Santa Monica, California, USA. Kite is engaged in the development of innovative cancer immunotherapies. The company is focused on chimeric antigen receptor and T cell receptor engineered cell therapies.
 

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.

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.

VIVET Therapeutics

Vivet Therapeutics is a clinical-stage biotechnology company developing novel gene therapy treatments for rare, inherited metabolic diseases.
 
Vivet’s lead program, VTX-801, is currently under clinical development for Wilson Disease (GATEWAY – Phase 1/2 Clinical Trial of VTX-801).
 
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) and Fast Track designation by the FDA. Wilson Disease, a 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 recombinant adeno-associated virus (rAAV) and is also working on technological platforms addressing key challenges of gene therapy, including sustained therapeutic gene expression in young patients, adults and immune responses towards the viral vector.