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.
 

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

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. We routinely post information that may be important to investors on our website at pfizer.com.

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.

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.

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.
 

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.
 

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.
 
 

Polyplus-transfection

Polyplus-transfection applies its 15+ year expertise to the development of novel transfection solutions up to GMP grade for high yield transient protein and antibody production in CHO and HEK-293 cells, as well as for viral vector production for Gene and Cell Therapy (PEIpro product range).

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.

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.

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.