Cell and gene therapies are a new frontier in medicine. Rapid developments in this field have the potential to change the way patients with cancer 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.
Cell and gene continues to be an area with rapid growth in investment and product pipelines. In 2021, the cell and gene therapy sector has seen strong clinical pipeline growth, commercial progress and record breaking investment. As of end of 2021, globally there were more than 2,700 cell and gene therapy products in development from pre-clinical through pre-registration stages1. The sector had the highest annual number of regulatory approvals of new cell and gene therapy products1. In terms of investment, in 2021 the sector received the highest annual financing to date. Companies in the sector raised more than $23 billion, which is 16% increase from 20202.
There is a clear increase in the portfolio of therapies. The CDMO market outlook for Cell & Gene Technologies (CGT) shows mid-teens growth for the pre-clinical and early phases. This increases to the mid-twenties for the late-stage and commercial phases3. A compound annual growth rate for the whole CGT CDMO market is expected to reach more than 15% (CAGR 2021-2023)3. As customers gain greater visibility of the potential commercial success of their therapy, there is a greater appetite for longer contracts, which provide benefits to both customer and supplier.
The manufacture of these treatments also brings new challenges that range from the need for specialized media and other critical raw materials to gene-editing tools and IT. For example, the small patient-scale batch sizes for autologous products require automated solutions. This is critical 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 and requires stringent data integrity and vein-to-vein traceability. This is driving an increasing need for cost-effective and flexible IT systems, which can be rapidly deployed to improve decision making, quality and compliance needs. For allogeneic cell and viral vector gene therapies, there is a challenge in scaling-up and optimizing processes to increase yields and treat more patients per batch, while continuing to meet highest quality standards.
The cost of production still represents a major hurdle on the path to commercialization. New technologies that enable robust and efficient manufacturing to achieve replicable and high-quality treatments will be a cornerstone in the growth of the cell and gene therapy market. Innovation in manufacturing will be essential for the long-term success of bringing these therapies to patients.
Nonetheless, market growth is driven by improved clinical efficiency, allowing an increasing number of products to move towards late-stage and commercial phases, supported by accelerated approval pathways. Looking across the industry, the high potential of the market is becoming increasingly clear.
ASGCT Gene, Cell, & RNA Therapy Landscape Q4 2021 Quarterly Data report
ARM’s virtual 2022 Cell & Gene State of the Industry Briefing, January 10th 2022
2021-2023 CAGR for CGT Market in USD; Source: Informa Citeline and Lonza internal analysis
Our Cell & Gene divisional portfolio is concentrated around three business areas: Cell & Gene Technologies, Personalized Medicine and Bioscience. As we work to address the complexities in research, development and manufacturing, the division looks set to transform the way we treat patients with cancer or genetic diseases. By providing the critical raw materials and enabling technologies together with expertise to support the development and commercialization of innovative therapies, we help de-risk and accelerate the path to market.
cGMP Cell & Gene Technologies Experience
Process Development Projects
Primary Cell Types
Bioscience Products Filed with Regulatory Agencies
The Cell & Gene Technologies (CGT) business 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).
We provide an integrated offering of key services beyond traditional manufacturing, to meet customer needs end-to-end. These services include:
We also provide a service offering and expertise in emerging and promising cell and gene therapy modalities such as:
Personalized Medicine is a start-up business unit developing breakthrough technologies to industrialize autologous cell therapies. A prominent part of this business is our Cocoon® Platform, a closed, automated system for patient-scale cell therapy manufacturing.
Our Bioscience business is a provider of specialty raw materials and enabling technology solutions. We provide our customers with the tools to develop, manufacture and test therapeutics, covering the entire journey to market, from gene to patient. Serving customer communities across academia, biotech and pharma, we can support all therapeutic modalities including cell and gene therapies, injectable drugs, vaccines and bio-manufacturing.
With our global network spanning three continents, we are supporting customers from research through to commercial production.
Facility owned and operated by Nikon Cell innovation Co. Ltd. under Nikon-Lonza partnership
Across the Cell & Gene division in 2021, there was strong customer demand. The business saw improved synergies between the Bioscience business unit and our CDMO services, such as increased media and buffer supply to biologics programs and growing interest in technologies such as Nucleofector® for cell and gene therapies.
Our business priorities are focused on driving profitable growth in Cell & Gene Technologies, strengthening the Bioscience offering for the cell & gene market, driving the adoption and commercialization of the Cocoon® Platform and further accelerating the synergies between the three business areas.
Comparison vs. Prior Year
CORE EBITDA (CHF)
CORE EBITDA Margin
Sales growth, expressed as a percentage (%), is at constant exchange rate (CER)
As anticipated, the Cell & Gene Technologies business achieved a positive margin in Q4 2021. We will continue to work towards securing long-term sustainable and profitable growth. With an increase in the volumes of customer products as they progress towards commercialization, we expect to see fewer changeovers at our facilities. This will also help in achieving more established production processes and higher asset utilization rates, which will in turn improve our operational efficiency and reduce our costs.
In 2021 we continued to invest in innovative modalities by expanding our Exosomes manufacturing offering with Codiak Bioscience’s exosome manufacturing site in Lexington (US) and Exosomics’ Service Unit in Siena (IT). Exosomes are emerging as a new modality for advanced therapies and could become the next frontier in biotherapeutics.
Our collaboration with Codiak, one of the most advanced companies in this modality will help drive the growth of the whole industry. The acquisition provides for worldwide access and sub-licensable rights to Codiak's high-throughput perfusion-based cGMP process, which we will make available to all Lonza customers for their exosome manufacturing needs. A Center of Excellence, established jointly, will leverage the strengths of both companies to advance developments in exosome production, purification and analytics.
With the addition of the Exosomics’ Service Unit in Siena (IT) to our network, we have gained access to experienced talent, state-of-the-art knowledge and the ability to advance this therapeutic area further. Under the terms of the agreement, we will gain access to expertise and capabilities in the exosome field including analytics and characterization.
Building on the success of the FDA Pre-Approval Inspection (PAI) completed in 2021 in Houston, we further established ourselves as the partner of choice for the late-stage clinical and commercial manufacturing of cell and gene therapies. Our track record in the cell and gene therapy space has the capacity to bring hope to many patients. In 2021, we announced an agreement with Aruvant Sciences to manufacture its gene therapy, ARU-1801, a potential cure for sickle cell disease that can be given with one low dose of chemotherapy. We have already started process development and technology transfer activities at our Houston (US) site.
Our collaboration with PsiVac is another example of how cell and gene therapies are paving the way for more options to treat serious diseases. We have been granted the exclusive rights to manufacture Ixovex-1, a unique, patented oncolytic virus designed to provide personalized therapy for cancer patients. Process development activities are under way at our Houston facility, with a Phase 1 clinical trial planned for Q2 2022.
President, Biologics and Cell & Gene Divisions
The cell and gene industry continues to grow at an accelerated pace. As more programs are approved, reliability and quality is critical to bringing treatments to market. To meet our customers’ needs, we have continued to invest to grow our network and capabilities, strengthen execution and differentiate through innovation.
Exosomes are emerging as a new modality for advanced therapies. The addition of two new sites in Lexington (US) and Siena (IT) has enabled us to enhance our extensive offer in this space and enable the manufacturing of new therapies.
In 2021, our proprietary Cocoon® Platform was used in a clinical trial with Triumvira where the first patient has been dosed with TAC-T cell breast cancer treatment. This development brings patient-scale therapy to solid tumors and has the potential to significantly improve clinical outcomes for some breast cancer patients. The Cocoon® Platform brings scalability, reliability and cost effectiveness at point of care.
We have also improved synergies between our Bioscience media business and our CDMO services. This has helped us to meet the demand for shorter timeframes for media and buffers, specifically with Moderna and Ibex® Solutions. Intercompany media sales have more than doubled and we have now started to co-develop media across some of our businesses.
Looking ahead to 2022, we plan to further develop our portfolio of services and products. We will continue to integrate the two new exosomes sites into our organization, and establish our position as a partner of choice for the late-stage clinical and commercial manufacturing of cell and gene therapies. This work will build on the success of the 2021 Pre-Approval Inspection (PAI) at our site in Houston (US).
Building on our experience, the Cocoon® Platform is already allowing us to tackle the traditional challenges of autologous cell therapy. We aim to enable our partners to provide personalized immunotherapies to critically ill patients faster, at a higher quality and lower cost. As we continued the commercialization efforts of the Cocoon® Platform during 2021, we entered into several collaborations with partners who are eager to establish a reliable, efficient and cost effective manufacturing process for scaling their therapies to market.
As part of our agreement with Triumvira, we successfully manufactured a TAC-T cell therapy treatment for a breast cancer patient. By transferring Triumvira’s TAC-T cell treatment to our Cocoon® Platform, the company was able to accelerate development efforts and achieve IND approval in less than a year. This collaboration showed the potential of the Cocoon® Platform to manufacture therapies at third-party manufacturing sites using a decentralized manufacturing model. Our collaboration with Leucid Bio is another example where the Cocoon® Platform will be used in a decentralized model to optimize and streamline the manufacturing process for Leucid’s CAR-T therapies.
The Cocoon® Platform is also demonstrating significant advantages in point-of-care manufacturing, as illustrated by our collaboration with CellPoint. The partnership is aimed at developing CellPoint’s T-cell therapies for treating various cancers using our proprietary Cocoon® Platform, alongside our expertise in process development and regulatory approvals in several EU countries. Throughout 2021, we have been working with CellPoint to develop a manufacturing process that will allow a six- to seven-day vein-to-vein time frame to treat cancer patients. We also continued our collaboration with the Sheba Medical Center in Israel, where the Cocoon® Platform was used to deliver CAR-T cell immunotherapy treatments.
As we continue to commercialize the Cocoon® Platform, we will expand our collaborations with leading research institutes and academic clinical centers, including Stanford University, Fred Hutchinson and Parker Institute.
Moving forward, we will maintain our focus on building additional capability and functionality into the platform to address unmet market needs, while ensuring 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 Cocoon’s high market potential.
2021 marked the 20th anniversary of our Nucleofector® Technology and the launch of the next-generation 4D-Nucleofector® Platform. As molecular biology comes of age in medicine, our Nucleofector® technology is used increasingly to introduce DNA, RNA, proteins and other molecules such as CRISPR/Cas9 into cells for research and development of therapeutics. The device has been cited in more than 10,000 peer-reviewed publications since its introduction, clearly demonstrating its importance to researchers around the world. The next-generation 4D-Nucleofector® Platform brings user experience (UX) improvements that make the system even more intuitive and easy to use, firmly securing its place as a core method in cell-based research. Similar enhancements are planned for the high-throughput Nucleofection systems.
Throughout the year, we leveraged our expertise to develop new products that support the cell and gene space. We expanded our offering with high-quality cryopreserved Leukopaks, which allow for more flexibility in immunology and cell therapy research.
Bioscience testing products have also supported the safe release of COVID-19 vaccines, resulting in increased sales as many companies scaled up manufacturing. Disruption caused by the pandemic focused efforts and investment on digital transformation projects, with increased interest in the implementation of electronic batch records and opportunities for our MODA-ES® Platform.
The pandemic has also raised awareness of the need for sustainable endotoxin testing. We have expanded our PyroTec® PRO Automated Robotic Solution for endotoxin testing to include options for rFC (a synthetic alternative to horseshoe crab-derived LAL tests). Through process optimization and automation of routine manual tasks, the PyroTec® PRO Automated Robotic Solution enables users to streamline and improve the performance of the QC laboratory, increasing lab efficiency and productivity.
Cell therapies, particularly patient specific (autologous) gene modified cell therapies, continue to flourish. The recent commercial approvals of gene modified cell therapies have shown incredible promise for the treatment of numerous oncological indications. However, the cost of these therapies will need to reduce significantly if this treatment paradigm is going to be competitive in the long term. Other biologics, or allogenic derived cell therapies, are possible alternative treatment options that have shown promise in early clinical trials and can potentially be provided at a much lower cost.
The key driver of cost for these gene modified cell therapies is manufacturing. Current manufacturing is labor intensive and cannot be scaled efficiently. We introduced the Cocoon® Platform in 2020 to address these limitations with the manufacturing of patient-scale cell therapies. The Cocoon® Platform is a closed and automated patient-scale bioreactor that performs the majority of the steps in the process. By closing and automating the manufacturing process, labor and facility costs are significantly reduced while quality and consistency are improved, leading to lower rates of batch deviations and failures.
In addition, the Cocoon® Platform provides the opportunity to add additional analytical capabilities to more closely monitor and optimize the manufacturing process. By partnering with companies like Agilent, parameters like cell fitness and potential can be identified. This allows for real time adjustments to the manufacturing process, which may ultimately lead to a more potent and effective product. Automation is the only way that patient scale cell therapies can be manufactured at a cost that will be competitive in the long term. The Cocoon® Platform is leading the way in this field and is poised to be a leading solution, as the market continues to expand.