With almost 2,8001 products in development across the industry, the cell and gene therapy sector has seen tremendous growth and interest over the last few years. Novel treatment candidates demonstrate the potential to change the way patients with cancer and genetic diseases are treated.
In 2022, the market saw clinical pipeline growth. Autologous products make up around 40% of the cell and gene therapy pipeline, followed by allogeneic products (around 30%) and in vivo viral vector products (around 30%)1.
While the autologous cell therapy area grew significantly in the last five years, it is showing signs of slowing down, partly due to the impact of the COVID-19 pandemic on patient treatment. Nonetheless, while allogeneic cell therapy surpassed other modalities in 2022, autologous cell therapy is expected to remain the primary market category in the near future.
Immune cell-based therapies dominate the market, with T-cells and natural killer (NK) cell products driving interest due to their potential to address potential bottlenecks of autologous cell therapies, including cost-of-goods, scale-up efforts, and the ability to ramp up or down based on demand. Viral vector continues to benefit from healthy market growth both as a therapy and raw material.
There is a developing market tension as funding across the biotech industry is decreasing while competition in rare disease areas is increasing. In this context, speed to clinic and market is set to become even more critical, alongside access to complex manufacturing technology, safety and efficacy.
Regulators are paying increasing attention to the cell and gene therapy space. Despite accelerated pathways, cell and gene therapies remain subject to the same approval processes as traditional biologics. Ensuring safety and efficacy is not the only focus. Therapy developers also need to demonstrate the mechanism of action, process robustness, scalability and potency of their drug candidates. In response to these imperatives, process and analytical development are expected to play an increasingly important role towards commercialization.
The cost of production remains a significant hurdle on the path to commercialization. This means that investing in platforms that increase productivity and offer flexible manufacturing ramp-up or ramp-down in response to demand is becoming more important to success. As a result, companies need to consider commercialization challenges from the early phases of drug development.
New emerging modalities and tools continue to grow within the cell and gene therapy sector, including exosomes, induced pluripotent stem cell (iPSC)-based immunotherapies, allogeneic versions of autologous CAR T-cells, NK cells, and in vivo gene editing. Continuing innovation in therapy development and manufacturing will be essential for long-term success and commercial viability. In this context, CDMOs are likely to take a more prominent role in the path to commercialization, supported by robust quality systems, expertise and an accelerated approach to scalable manufacturing.
Citeline Pharmaprojects Pipeline Search July 1, 2022, internal analysis
Our broad offering includes development and manufacturing services, products, solutions, testing and automation platforms. We also offer tools and technologies to enable our customers to develop, de-risk and industrialize therapies, from basic research to commercialization.
Our Cell & Gene division includes three business areas: Cell & Gene Technologies, Personalized Medicine, and Bioscience.
Years of C&G cGMP manufacturing experience
Process Development Projects
pre-clinical therapies supported by Bioscience
clinical and commercial therapies supported by Bioscience
Cell & Gene Technologies (CGT) is focused on providing an integrated range of CDMO services that span the full value chain of cell and gene therapy modalities (allogeneic and autologous therapies and viral vector).
Our integrated service proposition relies on two core pillars of CDMO services:
Additional CDMO services include:
We provide these service offerings across a wide range of modalities to bring our customers the expertise they need for their therapies. These include:
The Personalized Medicine business develops breakthrough technologies to accelerate the industrialization of cell and gene therapy manufacturing. A primary focus is our Cocoon® Platform, a functionally closed, highly flexible and scalable cell manufacturing solution. The Cocoon® integrates multiple unit operations including isolation, cell selection, activation, transduction/transfection, expansion, and harvest into a single system. This degree of process automation has the potential to drive down costs and provide greater access to patients. The Cocoon® Platform is commercially available and being deployed across a number of clinical programs in centralized and point of care manufacturing settings.
Our Bioscience business provides a range of solutions for customers working at different stages of the therapeutic journey across multiple modalities, from cell & gene therapies to recombinant proteins, vaccines, and injectable drugs. Our expertise in primary human cell biology tools enable customers to develop more predictive models and accelerate the path to IND. We are also a trusted, committed partner for critical raw materials and technologies that enable better processes and quality decisions for bioprocessing customers.
Our offering includes:
Our offering includes development and manufacturing services, products, solutions, testing and automation platforms. We also offer tools and technologies to enable cell and gene innovators to develop, de-risk and industrialize their therapies. We support customers from research to commercial production through our global network spanning three continents.
Facility owned and operated by Nikon Cell innovation Co. Ltd. under Nikon-Lonza partnership
In 2022, the Cell & Gene division benefitted from strong overall performance in the Bioscience business unit.
In our Cell & Gene Technologies business, two additional cell and gene therapies manufactured at our Houston (US) site achieved FDA approval. However, delays in clinical trials and customer product challenges impacted sales growth.
Our Personalized Medicines business unit remained focused on key R&D initiatives and scaling manufacturing, with multiple clinical-stage therapies now being manufactured on the Cocoon® Platform.
The Bioscience business continues to experience strong customer demand across its portfolio, especially in testing and media. A strategic reconfiguration in Bioscience in 2022 will support the business to deliver innovative products at scale in the long-term, with an emphasis on capturing market share in growth modalities, specifically cell and gene therapies and next-generation biologics.
Comparison vs. Prior Year
CORE EBITDA (CHF)
CORE EBITDA Margin
Sales growth is at constant exchange rate (CER)
Our site in Houston (US) is dedicated to cell and gene therapy development and manufacturing. In Q3 2022, two cell and gene therapies manufactured at the site were approved by the FDA for commercial use, demonstrating our continued focus on improving quality and operations in collaboration with regulatory authorities. ZYNTEGLO®, for the treatment of transfusion-dependent beta-thalassemia, and SKYSONA®, for the treatment of early, active cerebral adrenoleukodystrophy, are both produced by bluebird bio of Somerville, Massachusetts and were approved in August and September 2022, respectively.
Also in 2022, the CGT business enhanced the New Product Introduction (NPI) process to standardize the customer journey from early-stage development to commercialization. It provides a roadmap and a systematic approach to development and manufacturing, ensuring necessary quality standards are met for tech transfers, cGMP manufacturing and pre-approval inspection readiness. This NPI process has been designed to support more CGT customers in reaching commercialization.
President, Cell & Gene Division
Since joining Lonza in November 2022, I have been impressed with the focus on meeting customers’ needs and driving technological innovation across the Cell & Gene division.
In our Cell & Gene Technologies business unit, our people are unlocking opportunities to capture the high commercial and therapeutic potential of this rapidly growing market. Following commercial approvals for two therapies produced at our Houston (US) site in 2022, we now manufacture three commercially available cell and gene products. We have also invested to expand our process development capabilities at our laboratories in Houston (US) and Geleen (NL) to meet evolving customer needs.
While we continue to see healthy growth in the clinical pipeline, there has been some reduction in the availability of biotech funding. In this context, operational excellence has become increasingly important, to ensure that value is optimized through efficiency. To support our customers in this area, we continue to focus on driving continuous improvement to enhance delivery and quality across our network.
As I commence my first full year with Lonza in 2023, I am greatly looking forward to leading the Cell & Gene division into its next chapter of growth. Our key priority for the coming year is to build our pipeline to support the development and commercialization of innovative therapies. We will continue to offer customers an integrated and accelerated approach to project scale-up, supported by robust quality systems and strong levels of technical expertise.
Through our Cocoon® Platform, we are addressing challenges traditionally associated with autologous cell therapy. The platform enables our partners to provide personalized immunotherapies to critically ill patients at a higher speed and quality, while managing the costs associated with such personalized treatments.
During 2022, we expanded the functionality of the Cocoon® Platform by releasing a second-generation instrument that includes integrated capabilities in cell binding, cell separation, and bead removal. The Magnetic Selection capability, which can be utilized at any point in the manufacturing process, provides a high level of customization and consistency and expands the end-to-end solution for cell therapy manufacturing. This innovative new functionality will further strengthen the Cocoon® Platform’s leading role in the commercialization of cell therapies. Ultimately, it will help to advance discoveries into the clinic where they can benefit patients.
Moving forward, we will maintain our focus on building additional capability and functionality into the platform to address unmet market needs, while continuing to ensure system robustness and exceptional customer service. Our goal is to build an autologous cell therapy manufacturing capability focused on cancer and monogenic rare diseases, while building further on the high market potential of Cocoon®.
Across the year, we leveraged our expertise to develop new products that support the cell and gene market. One example is the launch of the PyroCell® Monocyte Activation Test - Human Serum System (PyroCell® MAT HS System), which uses human serum instead of fetal bovine serum for in vitro pyrogen testing. This new system exhibits enhanced sensitivity for the detection of non-endotoxin pyrogens as well as reduced interferences from complex drug products such as biologics-based pharmaceuticals.
In early 2022, we began offering large batch sizes of human cord blood CD34+ hematopoietic stem cells for creating humanized mouse models. These are critical for the preclinical safety testing of immunotherapies. We are currently one of the leaders in the market with this offering. The additional offering will facilitate the more rapid and cost-effective creation of large and HLA-matched humanized mouse cohorts to streamline biologics testing and research.
In August 2022, we launched the Nebula® Multimode Reader, the first multimode reader qualified for use with Lonza’s turbidimetric, chromogenic and recombinant endotoxin detection methods. The new compact reader, designed to minimize laboratory footprint, is compatible with all of Lonza’s quantitative endotoxin tests and allows for an easier selection of the best-suited assay for specific samples. This addition to our portfolio complements our competitive advantage in QC automation.
The gene therapy field has achieved transformative progress over the past half-century, continually evolving to bring life-changing therapies to patients. Viral vectors lie at the heart of the field as a primary delivery vehicle of novel gene-based therapies. Recently, this area of the market has witnessed unprecedented growth, supported by a number of landmark regulatory approvals. This culminated in the recent regulatory approval of an adeno-associated viral vector (AAV)-based therapy for haemophilia B.
As a part of our holistic standardized process for developing and manufacturing AAV-based products, we have developed novel analytical techniques to ensure stability and quality of these vector-based therapies. These will help our customers to have more control of their product and better navigate the regulatory path to commercialization.
Our teams in Houston (US) and Basel (CH) have developed several analytical techniques targeting the in-depth analysis of AAV capsid proteins, viral genome, and AAV infectivity. These projects aim to develop accurate and robust analytical methods to ensure the quality and safety of AAV-based therapies for clinical applications.