Cell and gene technologies are seen as the new frontier in medicine. 2018 was a critical year for regenerative medicine financing, with investments of more than USD 13 billion1. In 2019, the level of venture capital (VC) financings already exceeded that of 20182. The influx of capital into the field of cell and gene therapy is testament to their potential to transform the way patients are treated, providing potentially curative or life changing patient outcomes.
Currently there are more than 1,000 regenerative medicine clinical trials2, with five landmark commercial approvals in the past two years. We are at an inflection point with an increasing number of products moving to late-stage and commercialization. By 2025, the US Food and Drug Administration (FDA) expects to approve 10 to 20 cell and gene therapy products per year3.
The manufacture of such medicines brings new challenges. For example, the small, patient-scale batch sizes for autologous products require automatized solutions to enable scalability and efficiencies in manufacturing to meet commercial demand for certain larger indications. Furthermore, getting these drugs to patients around the globe can present logistical challenges. For allogeneic cell and viral vector gene therapies, there is a challenge in scaling-up to increase batch sizes and treat more patients per batch. Today the cost of production still represents a major hurdle on the way to the market. New technologies must be developed to enable robust and efficient manufacturing and yield replicable high-quality medicines. These challenges need to be addressed to bring affordable curative medicines to patients globally.
1 Alliance of Regenerative Medicine (2018)
2 Alliance of Regenerative Medicine (2019)
3 Food and Drug Administration (2019)
We are at the forefront of this new frontier, with our comprehensive offering that spans products and services for allogeneic and autologous cell therapies, as well as viral vector gene therapies. Our competitive advantage resides in the combination of an integrated offering beyond manufacturing, with unmatched process development expertise and industrialization capabilities. We can offer in-house tissue acquisition, dedicated cell and gene therapy regulatory expertise, to an integrated supply chain orchestration system. Together, these services provide an end-to-end offering from concept to commercialization.
Years cGMP Experience
Employees (end of 2019 and growing)
Customers Over the Years
We enable our customers to de-risk their process early and fully industrialize their therapy via our unmatched experience in process development across cell and gene therapy modalities, supplemented with a full access to available technologies, all located under one roof in Houston, TX (USA). This provides customers with the best chance of success for commercialization.
We are already well-positioned with our extensive experience in cell processing, process development and manufacturing of cells and viral vectors under current good manufacturing practices (cGMP). Our tailored manufacturing solutions are built around a broad service offering including process development, bioanalytical services and global regulatory support. These offerings, combined with a global footprint spanning the United States, Europe and Asia, enable us to support our clients throughout clinical development and commercial production.
Our Modalities in Cell & Gene Technologies
Our Global Footprint
At our Houston, TX (USA) facility, we offer development and cGMP services for cell and gene therapies, including viral vector production. The range includes a wide selection of cell and virus types, including T-cells, dendritic cells, pluripotent stem cells (PSCs), mesenchymal stem cells (MSCs) and adenoviral, adeno-associated virus (AAV) and lentiviral vectors. Our Portsmouth, NH (USA) cell therapy cGMP provides clinical and commercial cGMP manufacturing.
In Europe, our dedicated cell and gene therapy manufacturing sites in Geleen and Maastricht (NL), provide capacity for both process development, analytical services, clinical and commercial cGMP manufacturing.
In Asia, we provide clinical and commercial cGMP cell therapy manufacturing from our Tuas (SG) site. We also partner with Nikon CeLL innovation Co., Ltd in Japan to provide our customers with process development, analytical services, clinical and commercial cGMP manufacturing specifically for the Japanese market.
Our global research and development (R&D) footprint is also growing. This is reflected in the ramping up of R&D capabilities in Houston, TX (USA), at the Israel Collaborative Innovation Center in Haifa (IL), and with the opening of R&D labs in Rockville, MD (USA).
Additional information about our services, such as process development, cGMP manufacturing, assay development, analytical and all other related services, is available on our Cell & Gene Technologies website.
Highlights and Initiatives 2019
During the reporting year, our Cell & Gene Technologies business benefitted from continued sales momentum in a dynamic market environment, with strong interest in offerings including process development and commercial manufacturing. We signed a significant number of clinical and commercial contracts with new customers.
Despite the strong commercial momentum, our Cell & Gene Technologies business is addressing the continuing need to improve productivity. The team is focused on improving contractual excellence to reflect the value of our services, optimize our network, and deploy meaningful operational excellence.
In 2020, the business expects at least five late-stage registrations within our global network. In the coming year, we will continue to focus on improving operational excellence and on delivering seamless service, removing supply chain challenges in autologous cell therapy and ensuring patient safety in personalized therapies while working on a “vein-to-vein” offering.
Clinical and Commercial Programs
During 2019, there was a strong interest in our offerings, including process development and commercial manufacturing capacity with new customers signed. We have signed a manufacturing service agreement with Cellectis for our Geleen (NL) site, for the clinical manufacturing of Cellectis’ allogeneic UCART product candidates targeting hematological malignancies. We have also established a strategic collaboration with Prevail Therapeutics, for its pipeline of novel AAV-based gene therapy programs to be developed and manufactured at our Houston, TX (USA) site.
Another example includes the strategic collaboration between Lonza and DiNAQOR AG to advance DiNAQOR’s preclinical programs for the treatment of cardiac myosin-binding protein-C (MYBPC3) cardiomyopathies, a genetic condition that can result in heart failure. The process development and manufacturing will be located at our Houston site.
In addition to these new partnerships, we have expanded existing collaborations with our long-term partners — Gamida Cell and Mesoblast. We have established a commercial manufacturing agreement with Gamida Cell for Omidubicel, a Phase III investigational advanced cell therapy designed to enhance the life-saving benefits of hematopoietic stem cell (bone marrow) transplant. Under this multi-year agreement, we will construct dedicated production suites at our Geleen (NL) site, for the anticipated commercial launch.
We announced the signing of a commercial manufacturing agreement with Mesoblast for the production of MSC-100-IV, a mesenchymal stem cell-based therapy pending commercial approval by the Food and Drug Administration (FDA) for steroid refractory acute graft versus host disease. The two companies have formed a strategic alliance since 2011 for the clinical and long-term commercial production of Mesoblast’s off-the-shelf (allogeneic) Mesenchymal Precursor Cell portfolio of products. Production of MSC-100-IV will be carried out in the existing suite in our cGMP facility in Tuas (SG).
Pursuing the goal of seamless service for our customers and their patients, we announced two partnerships to enable a ‘vein-to-vein’ delivery network. Vineti will provide a supply chain orchestration platform that allows easy access to fully electronic end-to-end control of material, and reduce the time that biopharmaceutical developers need for system selection and integration. Cryoport will provide transport and delivery of patient tissues on a global basis, ensuring seamless service for our customers and their patients. We will work with these two new partners to remove the supply chain hurdles faced by developers of personalized therapeutics.
In March, we started to bring our CocoonTM autologous cell therapy in-a-box manufacturing device to the clinic as a pilot project with Sheba Medical Center, the largest hospital in Israel. This collaboration is a key part of the development program for the CocoonTM platform. It will confirm the benefits of using our closed, automated “cGMP-in-a-box” concept to more efficiently manufacture personalized cell therapies where the patients need it. This will enable treatment of a larger patient population.