Realising the full curative potential of cell therapy
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Cell therapy is a rapidly advancing field offering the potential for curative treatments across disease areas with significant unmet need.
What is cell therapy?
Cell therapy refers to the use of engineered cells to prevent or treat a disease.
- The most established approach is autologous chimeric antigen receptor T-cells (CAR Ts). This involves the genetic modification of a patient’s own immune cells – such as T-cells, which are critical to the body’s natural defence mechanisms – so they can target disease-causing cells when reinfused as a bespoke medicine. The creation of autologous CAR Ts requires a highly specialised infrastructure, including dedicated manufacturing facilities and treatment centres.
- Allogeneic cell therapies are created in a laboratory from a healthy donor’s T-cells1
- In vivo cell therapies use viral vectors to deliver genetic instructions and reprogramme T-cells directly within the body.2
Jump to section:
- Our cell therapy ambition
- AstraZeneca’s latest updates in cell therapy
- Pioneering innovative cell therapies that target underlying drivers of disease
- What approaches is AstraZeneca exploring in oncology and haematology?
- Aiming to reset the immune system in immune-mediated diseases
- Engineering next-generation cell therapies
- Scaling manufacturing and clinical capabilities to bring cell therapies to more patients
- Enhancing our world-leading capabilities through external collaboration
- Open innovation
- Join us
Our cell therapy ambition
Our ambition is to realise the full curative potential of cell therapy across a wide range of diseases. We aim to empower the immune system to attack cancers, reset the underlying drivers of immune-mediated diseases, and deliver innovative solutions with the potential to improve the lives of people living with rare diseases.
To achieve this, we are building world-class cell therapy capabilities and advancing a diverse pipeline spanning CAR Ts, T-cell receptor therapies (TCR T), CAR T regulatory cells (CAR Tregs) and in vivo CAR T.
We strive to be at the forefront of pioneering the latest technologies to eliminate the barriers standing between patients and cell therapies. Through collaboration with industry, academia, healthcare and regulatory agencies we are committed to building an ecosystem that fosters access to cell therapies, to realise the full curative potential of these transformative medicines.
We’re building a diverse pipeline of cell therapies designed to overcome today’s toughest challenges — from persistence to scalability — and unlock new treatment possibilities across cancer, immune-mediated, and rare diseases.
Pioneering innovative cell therapies that target underlying drivers of disease
We’re advancing a broad range of cell types to potentially provide treatment options for more patients across a range of diseases.
- CAR T-cells: T-cells that are genetically engineered to recognise surface proteins on disease-causing cells and remove or modulate them.3
- TCR Ts: Created by engineering patients’ T-cells to express TCRs that can recognise cancer-specific intracellular proteins.4
- CAR Treg cells: Genetically modifying Treg cells – which are critical to preventing an overactive immune response – to identify and home in on specific organs affected by immune-mediated diseases.5
What approaches is AstraZeneca exploring in oncology and haematology?
Cell therapy is pivotal to our ambition to eliminate cancer as a cause of death and we aim to harness the immune system’s T-cells to recognise and respond to cancer more effectively.
Haematological cancers
Cell therapy has transformed outcomes for some people living with certain types of blood cancer.1 A core focus of our clinical research is on advancing potential new treatments with the goal of improving outcomes for people living with multiple myeloma, the second most common blood cancer. This includes our dual-targeting autologous CAR T designed using a rapid manufacturing process, that aims to reduce treatment waiting times and deliver potentially more effective CAR Ts.
Expanding cell therapies to solid tumours
Unlike in blood cancers, where freely circulating cancerous cells are accessible to cell therapies, the immune-suppressing tumour micro-environment of solid tumours poses a barrier to treatment.6 To overcome this, we are researching innovative strategies to ‘armour’ our cell therapies so they can resist the immunosuppressive effects of TGFβ, a cytokine that is highly expressed in many solid tumours and that limits the activity of immune cells. Our growing pipeline of CAR Ts target hard-to-treat solid tumours including prostate cancer and hepatocellular carcinoma.
We are also advancing TCR Ts, created by engineering a patients T-cell to express TCRs, to target intracellular proteins including common tumour-driver mutations such as p53 and KRAS.
Together, CAR Ts and TCR Ts unlock a breadth of targets in cancer biology, enabling us to advance a diverse pipeline of preclinical and clinical stage cell therapies across a range of tumours.
Aiming to reset the immune system in immune-mediated diseases
By harnessing the power of T-cells, we aim to correct the underlying immune dysfunction that drives immune-mediated diseases. By doing this, we could potentially reset the immune system to offer hope for long-lasting remission–a functional cure–with a single administration for certain patients. We are accelerating the development of two autologous cell therapy platforms in immune-mediated diseases: CAR T-cells and CAR Tregs.
CAR Ts
Emerging early data have shown the transformative potential of CAR T therapy in immune-mediated diseases, such as systemic lupus erythematosus.7 By targeting dysfunctional B-cells that drive lupus and related conditions, it may be possible to deliver long-lasting remission to patients.
Alexion, our Rare Disease unit, is building on these efforts to extend our work in cell therapy to address certain hard to treat immune-mediated rare conditions. By focusing on the core immunopathology—dysfunctional immune cells that produce pathogenic proteins—we aim to deliver lasting benefits for more patients and address high unmet need across diverse communities.
CAR Tregs
Regulatory T-cells (Tregs) are a T-cell subtype that can prevent an overactive immune response by regulating the activity of other cells.5 Working with our partner Quell Therapeutics, we are harnessing this capability with CAR Tregs to potentially transform the treatment of a wide range of immune-mediated diseases, including type 1 diabetes and inflammatory bowel disease.
By targeting the underlying drivers of immune-mediated diseases, we want to reset the immune system with cell therapies to move beyond symptom control and progress towards long-term remission and one day, potentially cure.
Engineering next-generation cell therapies
Our leading gene-editing, lentivirus and biologics engineering capabilities enable us to engineer next-generation cell therapies that could overcome some of the challenges that exist today. We are pioneering innovative approaches to deliver the next wave of cell therapies that could more precisely and effectively target disease and be produced at scale.
For example, lengthy manufacturing timelines are one of the most pressing challenges with autologous CAR T-cell therapy that can lead to suboptimal T-cell quality and activity, and delays in treatment. We are exploring new ways to shorten these timelines.
- FasTCAR is our autologous cell therapy manufacturing method that considerably shortens manufacturing timelines8,9 and aims to deliver higher quality T-cells and potentially more effective therapies, and reduce treatment wait times.
Looking to the future, we are committed to our goal to deliver ‘off-the-shelf’ solutions, including allogeneic and in vivo cell therapies, to increase access for patients worldwide. We envisage a future where physicians could select from libraries of patient-ready cell therapies matched to their disease.
Scaling manufacturing and clinical capabilities to bring cell therapies to more patients
We are building the infrastructure required to bring cell therapies to patients globally, and we continue to scale up our manufacturing and clinical capabilities and grow commercial expertise.
Manufacturing cell therapies
Manufacturing cell therapies requires specialised capabilities, and having the right technology, expertise and infrastructure is critical for translating research into effective cell therapy medicines.
We have expanded our manufacturing footprint, which now encompasses eight global locations. This includes our recent investment in a state-of-the-art facility in Rockville, Maryland, US, that will focus on manufacturing for critical cancer and immunology trials and future commercial supply in the US.
Clinical capabilities
We are enhancing our clinical capabilities through partnerships with renowned cell therapy treatment centres, including the Moffitt Cancer Center, to accelerate and expand access to cell therapy.
Additionally, investigator-initiated trials of our investigational cell therapies in China have enabled rapid generation of proof of concept across oncology and immune-mediated diseases.
Enhancing our world-leading capabilities through external collaboration
Partnerships
- Moffitt Cancer Center: Our collaboration with the Moffitt Cancer Center aims to accelerate our oncology cell therapy pipeline.
- AbelZeta: Accelerating the development of our armoured CAR Ts for solid tumours in China.
- Cellectis: Harnessing our combined cell therapy expertise, capabilities and differentiated gene-editing technology to discover and develop novel allogeneic therapies.
- Medical research council: We are co-funding a postdoctoral fellowship scheme with the MRC to support research into key topics relating to cell therapy including patient safety, delivery in the human body and interactions with the immune system.
- Quell: Using proprietary Treg cell engineering technology to fix cells in a stable suppressive regulatory phenotype and potentially restore immune tolerance in the long term. Initially focusing on type 1 diabetes and inflammatory bowel disease.
Acquisitions
- Gracell: Growing our R&D and manufacturing footprint in China, bringing in a novel dual-targeting CAR T therapy and a differentiated rapid autologous CAR T manufacturing platform: FasTCAR.
- Neogene: Adding expertise in the discovery, development and manufacturing of next-generation TCR Ts to our cell therapy capabilities, and offering a novel cell therapy approach for targeting cancers, including hard-to-treat solid tumours.
- EsoBiotec: Expanding our range of cell therapy platforms to include in vivo with EsoBiotec’s ENaBL lentiviral vector technology. This approach aims to eliminate the need for time-consuming processes involved in traditional cell therapy manufacturing, such as immune cell depletion, potentially making the administration of cell therapies in minutes through a single IV dose possible.
Open innovation
Through our Open Innovation programme, we share compounds, tools, technologies and expertise with the scientific community to advance drug discovery and development. We now have over 400 R&D collaborations—and counting, including collaborations in cell therapy.
Join us: Working together to advance cell therapy
We welcome committed, talented cell therapy scientists to join us on what promises to be one of the most exciting, stimulating and rewarding journeys in 21st-century medicine. We are proud of our progress, prepared for the challenges that lie ahead, and confident that, in the next five to 10 years, cell therapy will help improve the outlook for patients with some of today’s most serious and life-limiting diseases.
References:
1. American Society of Cell and Gene Therapy. Cell Therapy Basics. Available at: http://patienteducation.asgct.org/gene-therapy-101/cell-therapy-basics. Accessed November 2025.
2. Bui TA, et al. Advancements and challenges in developing in vivo CAR T cell therapies for cancer treatment. eBioMedicine. 2024;106:105266.
3. National Cancer Institute. CAR T Cells: Engineering Patients’ Immune Cells to Treat Their Cancers. Available at: http://www.cancer.gov/about-cancer/treatment/research/car-t-cells. Accessed November 2025.
4. Memorial Sloan Kettering Cancer Center. T Cell Receptor (TCR) Therapy. Available at: http://www.mskcc.org/cancer-care/diagnosis-treatment/cancer-treatments/immunotherapy/tcr-cell-therapy
5. Goswami TK, et al. Regulatory T cells (Tregs) and their therapeutic potential against autoimmune disorders–Advances and challenges. Hum Vaccin Immunother. 2022;18(1).
6. Fonkoua LAK, et al. CAR T cell therapy and the tumormicroenvironment: Current challenges and opportunities. Mol Ther Oncolytics. 2022;25:69-77.
7. Muller T, et al. CD19 CAR T-Cell Therapy in Autoimmune Disease - A Case Series with Follow-up. N Engl J Med. 2024;390(8):687-700.
8. Ceja MA, et al. CAR-T cell manufacturing: Major process parameters and next-generation strategies. J Exp Med. 2024;221(2):e20230903. doi: 10.1084/jem.20230903.
9. Yang J, et al. Next-day manufacture of a novel anti-CD19 CAR-T therapy for B-cell acute lymphoblastic leukemia: first-in-human clinical study. Blood Cancer J. 2022;12(7):104.
Veeva ID: Z4-78845
Date of preparation: November 2025