List of Gene Therapy Companies in Netherlands - 16

Amarna Therapeutics is a privately held, pre-clinical biotech company developing transformative, potentially curative gene therapies for a range of both rare and prevalent diseases. The company is pioneering the development of gene therapies based on simian virus 40 (SV40), a macaque polyoma virus to which humans are immunologically naïve. Amarna has created a proprietary production cell line (SuperVero™) that, for the first time, makes it possible to produce SV40-derived vectors suitable for therapeutic use. Combining SuperVero™ with its genetically engineered SVec viral vector, the company’s fully-integrated gene therapy platform is truly unique in its intrinsic capability to deliver transgenes without eliciting immune responses to the vector nor to the transgene product. Leveraging this ‘natural’ non-immunogenicity feature of its gene delivery system in humans, Amarna is developing a broad pipeline of safe, effective and durable gene therapies focused on genetic disorders, autoimmune diseases and chronic inflammation. Founded in 2008, Amarna’s head office is based in Leiden Bio Science Park, the Netherlands, with a research subsidiary located in Seville, Spain.

Bespoke promoters and enhancers, for any tissue, any organism. At Annogen, our vision is a future where safe and effective advanced therapies are specifically expressed in the right tissues with no off-target effects. We believe that this can be achieved by bespoke promoters (genetic switches) and genomic expression sites. Therefore, Annogen is on a mission towards becoming the leading expertise center for gene expression engineering in order to improve the safety and effectiveness of tomorrow's cell and gene therapies. Annogen leverages its proprietary SuRE™ technology to identify regulatory DNA elements to be used for controlled (therapeutic) gene expression for gene & cell therapy, as well as for recombinant protein and virus production. In addition, we develop service projects based on our proprietary AIM™ technology to identify favorable gene insertions and their expression levels for more than 100,000 integrations in parallel. These approaches typically enable big improvements in any biomedical strategy involving the (over-) expression of genes. Annogen was founded in 2017 by Dr. Joris van Arensbergen, who developed and published the SuRE™ technology as a post-doctoral fellow in the laboratory of Prof. Dr. Bas van Steensel at the world-renowned Netherlands Cancer Institute (NKI). Today, Annogen is the trusted partner for bespoke gene expression engineering for leading biopharmaceutical, agricultural and academic research centers globally.

DegenRx is a biotech company developing AAV-mediated gene therapy for the treatment of Alzheimer’s disease. Using Adeno-Associated-Viral (AAV) vectors we deliver genes in the brain that initiate the local production of therapeutic antibodies

We are Erasmus MC. Our roots lie in Rotterdam, a city and port of international standing. We are the most innovative university medical center in the Netherlands and one of the world’s leading centers of scientific research. We are committed to achieving a healthy population and pursuing excellence in healthcare through research and teaching. Day in, day out, our staff, volunteers and students work with passion and dedication to achieve everything we stand for: safe, first-rate healthcare for patients with complex issues, unusual and rare conditions or acute needs. But we also stand for top-quality teaching that attracts ambitious, inquisitive and talented students and seeks to answer the healthcare questions of tomorrow. And we stand for world-class scientific research that bolsters our understanding of diseases and disorders and helps to predict, treat and prevent them. We do our work based on our basic principles, the core values of Erasmus MC, 'responsible'​, 'connecting'​ and 'enterprising'​. We believe that we provide the best care, research and education if we are responsible, binding and enterprising.

Hybridize Therapeutics is a spin-off from the Leiden University Medical Center and is a therapeutic company focused on developing kidney-protecting modalities for untreated kidney diseases.

The Netherlands Center for the Clinical advancement of Cell and Gene Therapies (NecstGen), situated in Leiden, comprises of a state-of-the-art facility and expert knowledge for the development, production, and commercialization of Cell and Gene Therapies. NecstGen helps academic and industrial Cell & Gene Therapy developers to enter the clinical stage by bringing together the required expertise from R&D, development, production, QA, QC and regulatory affairs in a brand new 4,000 m2 development & GMP manufacturing facility. We enable the next generation of therapies by translating research programs into actionalbe health solutions for patients. Our service offering: A flexible Cell and Gene Therapy manufacturing model - Full contract manufacturing services by our expert team for Cell Therapy and Viral Vector - We manufacture Pre-clinical or toxicology study material, Phase I, Phase II clinical trial material - GMP rental for any mammalian cell-based therapy type, including Cell and Viral Vector based therapies - QC, QA, and QP services supporting our cleanroom rental - Hybrid support on a flexible basis where our team compliments yours Process and assay development for Cell Therapies and Viral Vector - Process design and optimisation - Scale-up to clinically relevant volumes - Automation and closed processes - Assay development for in-process controls, release- and potency tests - Technology transfer in and out with flexibility in-mind for the future progress of your manufacturing - Consultancy-based work to advise on decision making Talk to NecstGen today about your needs for: - Induced Pluripotent Stem Cells (iPSC) - Chimeric Antigen Receptor T-Cells (CAR-T), Tumor Infiltrating Lymphocytes (TIL) T-Cell Receptor Modified T-Cells (TCR-T) - Natural Killer (NK) Cells/CAR-NK Cells - Dendritic Cells (DC) - Mesenchymal Stromal Cells (MSC) - Lentivirus (LV) - Aden-associated virus (AAV) - Oncolytic viruses - Cleanroom rental for any mammalian cell-based therapy type

Neogene Therapeutics is a global, clinical-stage biotechnology company pioneering the discovery, development, and manufacturing of next-generation engineered T cell receptor (TCR) therapies targeting a broad spectrum of solid cancers. With offices in Santa Monica, CA and Amsterdam, the Netherlands, Neogene is aiming to change the paradigm of treatment for solid cancers. Neogene is a member of the AstraZeneca Group.

Imagine a world where we conquer cancer and genetic diseases. At NTrans Technologies, we solve key challenges in cell and gene therapy: Delivering life-changing therapies to the right cells and tissues. We leverage nature's own mechanisms for cellular uptake to ensure efficient and safe therapeutic delivery. Welcome to the next frontier of medicine. Our proprietary platform is GMP-ready and sets a new standard in cell and gene therapy. We use a non-viral delivery system to harness macropinocytosis - a natural cellular uptake process - for precise and secure delivery of gene-editing technologies. The mission of NTrans Technologies is to become the leading non-viral delivery platform in cell and gene therapy. Please contact us at: info@ntranstechnologies.com

Pharming Group N.V., a biopharmaceutical company, develops and commercialize protein replacement therapies and precision medicines for the treatment of rare diseases and unmet medical needs in the United States, Europe, and internationally. The company’s lead product is Ruconest, a recombinant human C1 esterase inhibitor that is used for the treatment of acute hereditary angioedema. It also engages in the development of rhC1INH for the treatment of pre-eclampsia, acute kidney injury, and COVID-19; leniolisib, a phosphoinositide 3-kinase delta (PI3K delta) to treat patients with activated PI3K delta syndrome; and alpha-glucosidase therapy for the treatment of pompe and fabry diseases. The company has a development collaboration and license agreement with Novartis; and a strategic collaboration agreement with Orchard Therapeutics plc for research, development, manufacturing, and commercialization of OTL-105, an investigational ex-vivo autologous hematopoietic stem cell gene therapy for the treatment of hereditary angioedema. Pharming Group N.V. is headquartered in Leiden, the Netherlands.

Phlox Therapeutics pioneers gene therapy to alleviate and cure cardiomyopathies. By embracing new ideas, we want to change the world for patients to whom the current standard of care falls short by leveraging RNA-based strategies to reduce the negative effects caused by genetic mutations.

We are ProQR, a biotechnology company dedicated to changing lives by developing RNA therapies for rare genetic diseases. We focus our drug development mainly on a group of blinding disorders affecting the retina, called inherited retinal diseases.

ProteoNic is a biotechnology company active in development and licensing of DNA technology for cell line generation and recombinant protein production for pharmaceutical applications. We offer premium vector technology that substantially boosts production levels of therapeutic proteins and other biologics. Cost savings are immediate and sizeable, freeing up production capacity and enabling commercially viable production of difficult to express proteins. ProteoNic's 2G UNic™ drives higher production for all types of therapeutic protein products, including monoclonal antibodies, biosimilars and more difficult-to-express (DTE) proteins, such as Fc-fusion proteins and bi-specifics, contributing to their economic viability. Our patented technology does not require modifications to existing production systems or work flows and is compatible with all production cell lines and expression systems used in the industry. Independent technical and commercial validation by third parties, including top tier biopharma, demonstrates severalfold productivity level increases that translate into multi-million Euros in annual cost savings. The technology has wide ranging potential to boost production efficiencies in other important applications, including viral vector production, protein production in yeast and technologies which impact product quality. The company is headquartered at the BioScience Park in Leiden, the Netherlands and has a Boston, USA office for Business Development in the North American market.

Syngle Therapeutics is focused on developing an immunotherapy for the treatment of Parkinson’s disease. Syngle possesses several antibodies against oligomeric alphasynuclein, the main toxic species in Parkinson pathology.

Treeway is a clinical-stage biotech company developing therapy for ALS, Alzheimer's Disease and other neurodegenerative diseases. Treeway has been formed by the words tree of life and pathway of a disease, for the founders the symbolic way towards a cure. We foresee that medication, research and care will change dramatically in the coming decades and patients will become an essential part of this process on their pursuit for cures in rare and common diseases. Everyone can make a difference and our slogan "one man can and will find a way, why not be that man?"​ underpins our drive.

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 severe genetic diseases.

VectorY combines the therapeutic potential of antibodies and gene therapy to develop long-lasting therapeutic solutions for large disease areas of high unmet medical need. By combining state-of-the-art technologies and novel and scientific concepts, VectorY’s mission is to bring innovative therapies to patients worldwide. Founded in August 2020, and based in the Amsterdam Science Park, VectorY is a fully integrated gene therapy company focused on the development of innovative therapeutics such as vectorized antibodies for both CNS and somatic disorders, with a special focus on muscle diseases. With R&D and manufacturing facilities in Amsterdam, VectorY develops proprietary & partnered programs based on its novel AAV-based vectorized antibody & gene therapy platform. Product candidates are based on new vector technologies, which will enable the next generation of highly scalable manufacturing processes within VectorY’s own manufacturing facilities. Our manufacturing capabilities will include a state-of-the-art multi-product GMP facility in the Netherlands, with the capability to deliver suspension based AAV viral vector manufacturing of up to 2000L for both clinical and commercial supply.