Our R&D focus areas

AstraZeneca’s Open Innovation program allows scientists from anywhere in the world to access our compound library and drug discovery platforms to support the discovery of new disease pathways and medicines

The path to new treatments

The future of treatment for many of today’s diseases lies in uncovering mechanisms that are newly emerging or still to be discovered. Focusing our intellect and resources in specific disease areas is the most effective manner to achieve truly breakthrough therapies. Our focus areas are Cardiovascular and Metabolic diseases; Oncology; Respiratory; Inflammation and Autoimmunity; and Neuroscience.

By exchanging resources and knowledge with leading scientists around the world, we aim to accelerate the discovery of novel therapies for unmet medical needs.

Partnering with academic and industry scientists outside AstraZeneca is critical. Combining our strengths and resources with the expertise and knowledge of our partners will ultimately benefit patients. We want partnering with us to be an easy and straightforward experience.

Kumar Srinivasan VP, Scientific Partnering & Alliances, IMED Biotech unit, AstraZeneca

Cardiovascular and Metabolic Diseases

Following the science to help people maintain and improve cardiovascular and metabolic health

AstraZeneca has a patient-centric approach to disease treatment, so we are tackling multiple risk factors by uniting our cardiovascular (CV), metabolic and chronic kidney disease (CKD) disease areas into one integrated approach – cardiovascular and metabolic diseases (CVMD). Our aim is to further reduce cardiovascular morbidity and mortality, and organ damage by addressing multiple CV risk factors.

Areas of research interest

Heart failure-Cardiac regeneration

We aim to deliver life-changing regenerative treatments to heart failure patients. By combining target validation on human stem cells with targeted delivery of small molecules and modified RNA, we seek to achieve activation of endogenous cardiac stem cells, induce cardiac tissue regeneration and improve heart function. Our ultimate goal is to be able to provide differentiated therapies to cure heart failure through disease modification.


Our scientific focus is to improve islet health and reverse non-alcoholic steatohepatitis (NASH). We are discovering novel therapies by using innovative technologies such as stem cells and genetics, to explore islet, adipocyte and liver biology. We emphasise new modalities, including miRNA, modRNA and antisense oligonucleotides. If successful, our research may one day result in unique treatments for pancreatic beta cell loss and NASH.

Chronic kidney disease (CKD)

We focus on delivering therapies to halt, slow or reverse the progression of kidney disease. We use human target validation to identify novel molecular pathways, and genetic and epigenetic methods to develop non-invasive prognostic biomarkers for patient stratification. If successful, such therapies have the potential to offer patient-centric approaches to treating kidney disease.

Collaborations with leading scientists allow us to generate data faster and to further define patient populations that may benefit from the novel cardio-metabolic therapies we are developing.

Marcus Schindler Head of the Cardiovascular and Metabolic Diseases Innovative Medicines Unit, AstraZeneca


Redefining the treatment paradigm to eliminate cancer as a cause of death

At AstraZeneca, we are working to redefine the cancer treatment paradigm by delivering life-changing medicines to help address unmet clinical needs in a host of cancers. We have strong franchises in lung, ovarian and breast cancer and we are building a haematology franchise. With a combination-focused pipeline that exploits the power of three scientific platforms, we are motivated by a dedication to scientific discovery and collaboration that will one day help eliminate cancer as a cause of death.

Areas of research interest

Genetic drivers and resistance mechanisms

In tumours with genetic drivers, potent inhibition of those drivers can lead to tumour shrinkage and improvement in progression-free survival. However, tumours eventually develop resistance to these mechanisms. We seek to target both genetic mutations and resistance mechanisms to drive towards more meaningful clinical improvements for patients.

Immune-mediated therapies

Recent data demonstrate that immune-mediated therapies can produce durable remissions and the possibility of a dramatic improvement in survival.  We are developing a broad portfolio of these agents for use as monotherapy, as well as combinations with other immune agents and drugs in our small molecule portfolio to exploit mechanistic synergies.

DNA damage response (DDR)

Halting cancer cell proliferation is only one component of tumour therapy; actively tipping cells into cell death is required for optimal clinical activity. Selectively targeting the DNA damage response, which plays a role in many sporadic and inherited cancers, is a key component of this approach. AstraZeneca has a portfolio that targets distinct aspects of DDR including PARP, ATR, ATM and WEE1.

AstraZeneca has a deep and broad oncology development programme to address unmet needs across a wide range of cancers. We have built a network of world-class collaborations and our open innovation research initiative complements our own efforts brilliantly.

Susan Galbraith Head of the Oncology Innovative Medicines Unit, AstraZeneca

Respiratory, Inflammation and Autoimmunity

Transforming disease management and patient outcomes

For 40 years, AstraZeneca has been pushing the boundaries of science in respiratory disease, and by following the science, we’re breaking new ground in inflammatory and autoimmune diseases. Through better understanding of the complexity of the immune system we aim transform disease management and patient outcomes beyond symptom control. By following disease drivers and their biological pathways we hope to change the natural course of diseases, with a primary focus on asthma, COPD and systemic lupus erythematosus (SLE). 

Areas of research interest


The unmet needs in the field are many and complex. The next generation of therapies must leverage the full array of genomic, molecular and biological insights and we believe addressing disease drivers are more relevant than looking at disease severity.

Our approach to the heterogeneity of these diseases is a scientific framework that maps out three central biological pathways to chronic obstructive and autoimmune conditions, aiming for targeted therapies with short and long term clinical benefits. Within these pathways, we focus on biological processes that may represent emerging “treatable traits” for complex lung diseases and autoimmunity, including:

  • Primary disease prevention and remission in T2 mucosal inflammation.
  • Defects in mucosal immunity that allow bacterial colonisation and promote vital exacerbations.
  • Arresting progression of premalignant epithelium towards lung cancer.
  • Epigenetic and stromal-somatic control of diseased tissue phenotype.
  • Macrophage lineage plasticity.
  • Shared molecular determinants of comorbid disease, especially heart disease and cancer.
  • Systemic autoimmunity.

The organising principle is to understand the fundamental drivers of diseases with large unmet medical needs such as asthma, COPD and lupus in subsets of patients and elucidate the associated disease pathways. This would enable application of an optimal set of mechanism-driven therapeutic strategies for each biological pathway and the various manifestations of disease arising from it.

Our top level aspiration is to move beyond the current era of highly effective symptomatic treatments to the next generation of medicines that can change the course of disease and bring about remissions and cures.

When science takes us beyond our core focus areas into other diseases, we may explore partnering routes and open innovation as a way of addressing unmet medical needs

Respiratory, inflammation and autoimmune diseases represent a main therapeutic area for AstraZeneca. Partnering with leading scientists helps to accelerate the development of drugs for patients who are suffering from these chronic conditions.

Maarten Kraan Head of the Respiratory, Inflammation and Autoimmune Innovative Medicines Unit, AstraZeneca


Advancing patient care in neurodegenerative diseases with unmet medical needs

AstraZeneca continues to push the boundaries of science in neuroscience in collaboration with other innovative partners across industry and academia. Our Neuroscience iMed focuses on the discovery and development of new treatments for psychiatric, neurological or analgesic disorders affecting the central or peripheral nervous system, across small molecules and biologics.

Areas of research interest

Neurodegenerative diseases

We are pursuing pathways and targets for multiple neurodegenerative diseases, including mitochondrial dysfunction, protein transmission, autophagy and neuroinflammation.  Disease interests include:

  • Alzheimer’s disease
  • Parkinson’s disease
  • Frontotemporal dementia
  • Huntington’s disease
  • Amyotrophic Lateral Sclerosis (ALS)


We are using recent advances in human genetics to identify targets and pathways for therapeutic intervention as well as capitalising on emerging biology around neuronal circuits known to be dysfunctional in disease. We focus on novel approaches to meeting high unmet needs in defined patient populations not treated effectively by existing therapies, including:

  • Autism spectrum disorders
  • Refractory major depressive disorder
  • Addiction disorders
  • Behavioural symptoms in dementia


We are focused on targeting pathways with potential for step-change efficacy compared with existing treatments, agents that provide robust efficacy in add-on settings and effectively treat non-responders to existing treatments. Our near-term approaches are aimed at:

  • Reducing afferent activation and transmission of nociceptive signals
  • Reducing peripheral sensitization
  • Preventing or reversing central sensitization
  • Modulation of signalling in spinal cord
  • Targeting descending control and affective components of pain.

We collaborate across academia and industry with the ultimate goal of developing new medicines as quickly as possible for people with neurological disorders or disease.

Iain Chessell Head of the Neuroscience Innovative Medicines Unit, AstraZeneca