Cell & Gene Technologies (CGT)

Market Trends

Cell and gene therapies are a new frontier in medicine; they have the potential to transform the way patients diagnosed with cancers or genetic diseases can be treated. These novel drug candidates have the capacity to provide improved patient outcomes and, in some cases, may even prove to be curative.

The cell and gene therapy market continues to grow at pace and – despite the global economic and social challenges of COVID-19 pandemic – 2020 proved to be a highly successful year for regenerative medicine. In 2020, the global financings in the sector broke the previous financing records that were made in 2018 (USD 13.3 billion) and reached USD 19.9  billion, showing a 100% increase from USD 9.8 billion in 2019 1. The influx of capital into the field of cell and gene therapy is testament to its potential to transform patient treatments and outcomes. Several pharmaceutical and biotechnology companies and academic institutions are developing cell and gene therapies to treat COVID-19. At the same time, there have been delays to the development of non-COVID-19 related cell and gene therapies, as supply chain lines and treatment centers have been impacted by the pandemic.

In 2020, the pipeline for cell and gene therapies continued to expand rapidly with more than 2,000 active therapies in development 2; there are more than 1,200 regenerative medicine clinical trials 1, with five landmark commercial approvals in the past two years of therapeutics for blood cancer, ocular disease, spinal muscular atrophy and blood disorder. We are at an inflection point with an increasing number of products moving towards late-stage and commercialization.

The manufacture of such medicines brings new challenges. For example, the small patient-scale batch sizes for autologous products require automated solutions to enable scalability and efficiencies in manufacturing to meet commercial demand for certain larger indications. Furthermore, getting these treatments 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 path to commercialization. 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. Source: Alliance for Regenerative Medicine (Jan 2021)

  2. Source: Alliance for Regenerative Medicine (H1 2020)

Our Offerings

Our vision is to enable customers to industrialize their process, from concept to patient. To achieve this objective, we have invested significantly in talent, resources and capacity across our global network of manufacturing sites in recent years. We have built state-of-the-art process development labs in Houston (USA) and Geleen/Maastricht (NL) to scale-up early stage processes and make them robust, replicable and commercially viable for our customers. In addition, we have increased our sites’ capabilities and expertise for current good manufacturing practices (cGMP). We now count over 1,000 employees worldwide in our Cell & Gene Technologies (CGT) business across our four centers of excellence located in Houston (USA), Portsmouth (USA), Geleen/Maastricht (NL) and Singapore (SG).

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Years cGMP Experience

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Process Development Projects

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Customers Served in 20+ Years

 

Centers of Excellence

Our offering within the CGT space stands out from the competition in three key areas:

  • We have an unmatched level of expertise and capability in process and assay development to successfully de-risk and industrialize processes from early stages to commercial scale. Our large and experienced process and assay development teams supported dozens of customers in 2020, enabling them to de-risk their process and advance towards market approval. These capabilities are coupled with global, state-of-the-art manufacturing across four sites on three continents.
  • We provide an integrated offering of key services beyond traditional manufacturing, to meet customer needs from end-to-end. This ability to support customers beyond manufacturing is a particular success factor for CGT customers due to its pioneering nature. Key services include:
    • Dedicated CGT regulatory support from initial regulatory submission to market authorization for fast-track approvals
    • In-house tissue acquisition services with customized solutions to navigate the complexities of tissue sourcing
    • Leading vein-to-vein partners for supply chain orchestration, apheresis network management, transport and logistics including Vineti, Cryoport and Be The Match BioTherapies
    • CGT media products, transfection technologies such as our proprietary Nucleofector® device & Bacterial Endotoxin Testing (BET)
  • We provide a service offering and expertise in emerging and promising cell and gene therapy modalities:
    • Exosome-based therapeutics manufacturing capabilities
    • Induced Pluripotent Stem Cell (iPSC) manufacturing expertise
    • Cell-based autologous and allogeneic immunotherapies manufacturing solutions
    • Viral vectors manufacturing for gene therapy including the production of adeno-associated virus (AAV), lentiviral and oncolytic viral vectors

Our Global Development and Manufacturing Footprint

With our global network spanning three continents, we have supported around 200 customers globally through clinical development and commercial production.

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  1. The facility is owned and operated by Nikon CeLL innovation Co. Ltd. under Nikon-Lonza Partnership

Highlights and Initiatives

In 2020, our Cell & Gene Technologies (CGT) business maintained business continuity through the pandemic, with strong demand for products and services. We reported strong sales growth, well ahead of the market. Margin and operational improvements were achieved, with increased throughput on existing assets 1.

Forecasts for 2021 anticipate further margin improvements in the coming year, as asset utilization continues to pick up and further efficiencies are achieved. Overall, cell and gene therapy market demand remains strong. In this context, we are confident that we can grow together with the industry at around 20% to 25% 2 annually for the next three years.

Our growth strategy is focused on early phase, pre-Investigational New Drug (IND) pipeline, and securing late stage clinical and commercial contracts. To support market demand and our growth, we are investing in new capacities across our site network. We continue to build capacity in Houston (USA) and Portsmouth (USA) while expanding in Geleen/Maastricht (NL).

To improve the efficiency in our production facilities, we have started implementing our proprietary MODA-ES® solution in our manufacturing centers, allowing patient traceability throughout the manufacturing process for our suites. In addition, the MODA-ES® allows open parallel processing of cell and gene therapies, which enables sites to improve production efficiency.

Personalized Medicine

Overall, 2020 has been a transformational and successful year for the Cocoon® Platform and for the Personalized Medicine business in general, and this is expected to continue into 2021.

As an important milestone, we qualified the Cocoon® Platform towards clinical and commercial readiness and treated the first patient at Sheba Medical Center (IL) with an autologous CAR-T therapy, which was manufactured using the Cocoon® Platform. The Israeli Ministry of Health approved use of the Cocoon® Platform to manufacture a CD19 CAR-T cell immunotherapy for an on-going Phase 2 clinical trial for B-cell malignancies after a full comparability study. The Cocoon® Platform will enable Sheba to reduce immunotherapy-manufacturing costs by lowering manpower, time and space requirements. This will also allow Sheba to deliver potentially curative cellular immunotherapies to more patients.

To enable the next generation of precision cell therapy manufacturing, we are collaborating with IsoPlexis on the use of the IsoLight automated proteomics platform. The platform will provide quality analytics for cell therapy products generated on our Cocoon® system. By using the Cocoon® Platform’s automated programming and on-board real-time analytics, the collaboration seeks to improve the biological understanding of starting material, and process and product analytics to enable more efficient manufacturing and higher quality cell therapies.

Another example of collaboration announced is with Noga Therapeutics to develop its autologous lentiviral gene therapy on the Cocoon® Platform. This collaboration will be the first use of the system to manufacture an autologous gene therapy aimed at curing a monogenetic gene disorder (immunodeficiency). The flexibility of the Cocoon® Platform and programming allow the system to be used for many processes, including this CD34 cell based gene therapy.

Further, we announced a series of additional collaborations, reflecting the crucial role of research institutes and academic clinical centers in developing next-generation cell therapies, including early-phase clinical cell therapy manufacturing. The collaborations include tech transferring cell therapy manufacturing processes developed independently at the Stanford University School of Medicine, Fred Hutchinson Cancer Research Center and Parker Institute for Cancer Immunotherapy onto the Cocoon® Platform. These leading institutions will evaluate the Cocoon® Platform’s potential to manufacture a range of unique cell therapies.

Clinical and Commercial Programs

In 2020, we announced a clinical manufacturing agreement, in place since 2018, with Rocket Pharmaceuticals for the development and manufacturing of Leukocyte Adhesion Deficiency-I (LAD-I). The announcement of the collaboration was made public following Rocket Pharma’s publication of positive preliminary data from Phase 1 and 2 trial of RP-L201 for LAD-I.  The agreement includes analytical assays and development services. Manufacturing takes place in both the Houston (USA) and Geleen (NL) sites.

To advance a next-generation, off-the-shelf, allogeneic immuno-oncology therapy, we signed a strategic partnership with Indapta Therapeutics. Indapta is developing a proprietary cancer therapy based on a specific, potent variety of Natural Killer immune cells for use in combination with multiple monoclonal antibodies. Within the collaboration, we will provide process development, clinical manufacturing and regulatory support for IND filing in our Houston (USA) site.

Over the course of 2020, we have committed to fighting the COVID-19 pandemic and deploying our expertise and resources to help our customers in this area. One example of our work is the agreement with Altimmune Inc regarding the manufacture of AdCOVID™, a single-dose intranasal vaccine candidate for COVID-19, designed to generate a broad immune response with the unique ability to promote nasal mucosal immunity. The agreement builds on existing collaborations with Altimmune and supports the customer’s commercial readiness to produce the vaccine in 2021.

Partnerships

Induced Pluripotent Stem Cells (iPSCs) are a critical material used in the development and manufacture of cell therapies, given their capacity to self-renew and their ability to differentiate into many different cell types. With the aim of making high quality iPSC therapies available at an affordable cost to our customers, we have entered into a worldwide agreement with FUJIFILM Cellular Dynamics, Inc. Under the agreement, FUJIFILM Cellular Dynamics grants us a non-exclusive right to use their patents related to iPSC generation, including episomal vectors and reprogramming factors, for the clinical manufacture and differentiation of iPSC lines in cell therapies. In return, we have granted FUJIFILM Cellular Dynamics a non-exclusive license for expanded use of our innovative Nucleofector® technology, to enable efficient transfection of cells, stem cell and cell lines. The agreement enables drug developers to leverage both companies’ expertise and technologies for the generation of iPSCs through licensing agreements.

In order to improve efficiency across the CGT supply chain and to reinforce our vein-to-vein network, we have announced a strategic partnership with Be The Match BioTherapies®. The partnership establishes Be The Match BioTherapies and Lonza as preferred partners and aims to support the companies’ shared goal of providing end-to-end solutions that streamline the development of cell and gene therapies across the CGT supply chain. This collaboration builds on existing partnerships announced by both companies including Lonza’s partnership with Cryoport and Vineti.

  1. Comparison versus 2019 at a constant exchange rate (CER)

  2. Revenue growth