List of Adeno-associated viral vector (AAV) Companies in Netherlands - 6

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.

Batavia Biosciences is a science-based CDMO that offers personalized services that fully support all the stages of biopharmaceutical development, from cloning to GMP manufacture and release of clinical material for phase I/II studies. We are not your standard CDMO - our customers regularly refer to us as their product development partner and thought partner. We focus on the early stages of product development with services ranging from DNA cloning, mammalian cell line generation, upstream process development, purification development, product characterization to clinical manufacturing. Headquartered in Leiden, The Netherlands, Batavia Biosciences is privileged to have strong strategic partners in both the EU and US. Batavia Biosciences aims to significantly contribute to ease human suffering from infectious disease and cancer.

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

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.

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.