Precision medicine

Targeting the right medicine to the right patient at the right time – a critical part of our approach to drug discovery.

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What is precision medicine?

Precision medicine is an innovative approach for tailoring disease treatment and prevention. It makes it possible for doctors and researchers to more accurately predict which treatments are more likely to work for a patient, taking into account individual genetic and molecular make-up, environment, and lifestyle.

By unravelling the complex underlying biology of many diseases, 开拓和应用先进技术, we are leading the way in the application of precision medicine. Our work, 以及澳门葡京赌博游戏与整个行业的合作伙伴的合作, biotech and academia, are driving for better treatments for patients as well as a more sustainable future for healthcare systems.

Find out how our BioPharmaceuticals teams are rising to the challenge of delivering precision medicines for chronic diseases

Our scientists are working hard to deliver the benefits of precision medicine. Using multi-omics, novel technologies, imaging, artificial intelligence, and machine learning they are digging deep into the biological processes that cause and drive disease. Our ambition is to develop and validate new treatments that make it possible to diagnose and intervene earlier, halt disease progression, achieve remission, and enable patients to have better outcomes and healthier futures across the world.



  • Identify novel drug targets that are expected to have a higher probability of success.
  • Identify biomarkers that can help characterise patients into subgroups most likely to benefit from treatment.
  • Support the design of better clinical trials, where the right patients are recruited to participate.
  • Develop diagnostic tests that can help guide treatment in the real world.

We’re applying a precision medicine approach across 90% of our R&D portfolio. 澳门葡京赌博游戏把病人放在医学研究的中心, confident that by matching the right treatment to the right patient at the right time, 在病情恶化之前进行干预, we can change the course of disease and allow patients to live better, healthier lives.



In respiratory diseases, like asthma, 人们对精确靶向刺激关键炎症途径的分子有着浓厚的兴趣,这可能导致未来新型精准药物的发展.

Suzanne Cohen Executive Director and Head of Bioscience Asthma, AstraZeneca

Asthma can be a devastating disease with 176 million attacks each year and debilitating symptoms, 尤其是那些患有更严重疾病的人.1


One example is our work to identify specific leukotrienes that drive inflammation, 疾病中支气管收缩和粘液的产生. 澳门葡京赌博游戏的精准医学研究正在探索这些分子作为尿液生物标志物,以帮助对患者的疾病进行分类,并将其与更有可能起作用的靶向治疗相匹配 to.

Chronic kidney disease

Chronic kidney disease (CKD)

Chronic Kidney Disease (CKD) encompasses various primary disorders and stages of progression, 患者群体是高度异质性的. The current symptom-based approach ignores the different underlying mechanisms. Our aim is to close this gap, by uncovering the underlying genetic and molecular drivers of disease to identify the right treatment for the right patient.

Pernille Laerkegaard Hansen Executive Director and Head of Bioscience Renal, AstraZeneca

Using unique datasets, the team has applied machine learning and artificial intelligence algorithms to classify patients into subclasses. For the first time, our research has shown that these disease categories based on molecular data are different from previous clinical classifications for CKD. We are now looking to identify urinary biomarkers which could be used to identify patients’ molecular disease classes non-invasively, allowing us greater precision when aligning the right patients to the right trials. In the future, our ambition is to provide tailored treatments based on individual disease, or patient, 由分子疾病驱动因素决定的类别.



In 慢性阻塞性肺病(COPD) we have seen little innovation in decades despite it being the third leading cause of death world-wide.2 To address this, we are dissecting the 500+ abnormally expressed genes and defining their role in disease. This precision medicine approach will help us identify potential novel targets for life-changing medicines.

Ken Grime Executive Director and Head of Bioscience COPD, AstraZeneca

通过靶向COPD的疾病驱动因素, such as oxidative stress, cell senescence, 慢性炎症和纤维化, we aim to develop precision medicines focused on disease modification so as to slow and stop disease progression. 为这些新疗法找到合适的病人, we need to understand more about COPD in different people and find new ways to assess the effects the disease. 从COPD患者队列中获得的数据为COPD的分子机制提供了新的见解,并帮助澳门葡京赌博游戏开发了非侵入性成像方法,这些方法可以在未来用于帮助患者分层或监测治疗反应. 在临床开发的早期阶段,将这样的精准医学方法结合起来,将有助于为后续的临床试验确定合适的患者.

Heart failure

Heart failure

In heart failure (HF), 患者可能有多种合并症, any of which may affect their outcome and response to heart failure therapies. To deliver our precision medicine approach we need to understand the molecular and genetic landscape, including the impact of comorbidities such as chronic kidney disease or obesity.

Kenny Hansson Executive Director and Head of Bioscience Cardiovascular, AstraZeneca

Targeting key mechanisms of HF including widespread inflammation, fibrosis, hypertrophy and microvascular dysfunction is a major priority.

Many HF cases have a hereditary component and by identifying variants in the genome we can gain new genetic insights. Among the genetic drivers in dilated cardiomyopathy is a mutation in the gene for phospholamban, which is linked to impaired heart muscle contraction and relaxation. This understanding enables the exploration of this genetic variant for the development of potential new therapeutic targets. 



Our precision medicine approach in metabolic-disorder associated steatohepatitis (MASH) is two-fold; firstly, we aim to identify the right patients with the presence of disease-causing gene variants, then we selectively disrupt this protein expression with novel antisense oligonucleotide (ASO) therapy.

David Baker Executive Director and Head of Bioscience Metabolism, AstraZeneca

MASH is a multi-component disease with high unmet needs and severe patient outcomes. 处于精准医学研究的前沿, 澳门葡京赌博游戏的目标是与MASH相关的基因突变, which are responsible for an approximately four-fold increase in risk of the disease.

For example, a single nucleotide substitution in the PNPLA3 gene severely impairs normal fat breakdown in liver cells. We are investigating ways to downregulate PNPLA3 and so potentially restore lipid metabolism.

Systemic lupus erythematosus


Our research is unlocking the science of the immune system to address significant unmet needs in Systemic Lupus Erythematosus (SLE). This complex disease is driven by multiple cell types and mediators, and we are exploring these with our precision medicine approach.

Tatiana Ort 澳门葡京网赌游戏生物科学免疫学执行董事

SLE is a chronic immune-driven disease in which the body’s immune system attacks healthy tissue in any part of the body.

At the forefront of our precision medicine approach is the Interferon (IFN) pathway which plays a central inflammatory role in SLE. 大约四分之三的SLE患者IFN基因特征升高,临床试验表明,过度表达与积极的治疗反应相关.

Harnessing this precision guided research could hold great potential to expand this approach into other conditions where, 尽管症状可能不同, 潜在的1型IFN基因标记是保守的. 


Our technology-driven approach is accelerating the way we design and develop new precision medicines and diagnostics tests, 推动科学发现的新时代.

Understanding the genome: Patient data banks are advancing our knowledge by helping to match genetic profiles and gene mutations to specific health outcomes. 基因组技术的不断进步使澳门葡京赌博游戏能够根据潜在的疾病机制将患者划分为亚组,并确定最合适的遗传靶点. 澳门葡京赌博游戏还对……进行了重大投资 multi-omic technologies (genomics, transcriptomics, proteomics, metabolomics, lipidomics) that are key to building a more complete picture of the complexities of disease, 哪些可以为新的测试和治疗提供信息.

Finding novel targets: 澳门葡京赌博游戏正在将澳门葡京赌博游戏丰富的数据集与外部患者数据来源相结合,并应用人工智能和机器学习来发现数据与疾病之间的关联, achieving healthcare breakthroughs more rapidly than ever before. For example, we are 与benevolentai合作 to create knowledge graphs which allow us to analyse vast amounts of scientific data to see potential interactions between gene targets, expression and disease.

Revolutionising imaging: Using advanced imaging technologies we can now capture at a molecular level the cellular interactions that can both define a disease, as well as monitor the efficacy of treatments based on the modulation of tissue biomarkers in response to a drug.

We are taking these advances in imaging technology into our clinical trials, redefining endpoints to demonstrate disease modification with targeted treatment.

A diagnostic case study with Almac Diagnostic Services in precision medicine for chronic diseases

加速设计新的诊断测试; Finding the right patients depends on reliable diagnostic tests. Ideally these should be capable of detecting early-stage disease and minimally invasive so they can be used to screen patients before symptoms develop.

We are collaborating with external partners to identify biomarkers and develop them into non-invasive tests, which could be validated for use in helping to diagnose patients.


Across the globe, healthcare systems are seeking new strategies manage the effects of a growing population living with complex disease comorbidities. 精准医疗为患者提供了明显的优势, healthcare systems and those involved in the provision of care – through earlier diagnosis, 避免不必要的治疗, 疾病过程的改善和更好的结果.


通过使治疗能够针对正确的患者, 精准医疗代表了一种新的医疗模式, evolving from the current untargeted approaches that often rely on ‘trial and error’ to find the best available treatment. 因为不太可能有效的治疗方法被避免了, there is less drug wastage, fewer side effects and total healthcare utilisation including length of hospital stays is reduced, providing savings to already stretched healthcare systems and building more sustainable care for everyone.

Precision Medicine FAQs


Precision medicine is an approach that helps to match the right patients to the right medicines at the right time. Typically, this involves using diagnostic tests that can be used to identify different groups of patients based on the biology of their illness. Right now, 病人通常根据他们的症状进行治疗, 这可能不能反映出他们患病的原因. In many cases this results in poor symptom control and does nothing to slow, 阻止或逆转疾病进展.

For example, standard medicines for people with asthma aim to prevent exacerbations and control symptoms but asthma can have many different causes. Precision medicine aims to provide diagnostic tests that will help doctors to learn more about the underlying causes of asthma in each patient. As a result of the tests, different groups of patients can then be given treatments that target the underlying causes of illness.




As a result, 精准医疗减少了诊断病人的猜测工作,这意味着更多的病人可以得到高效的治疗,不仅可以控制他们的症状,还可以帮助减缓病情, 阻止甚至逆转他们的疾病.


Precision medicine has the potential to transform healthcare. 由于精准医疗的目标是疾病的潜在生物学,因此有可能开发出更有效的治疗方法,并治疗目前没有选择的人. By incorporating diagnostics and specifically targeting the causes of disease, precision medicines also have the potential to detect more diseases earlier and to stop, or even reverse disease progression meaning that more people can get treatment sooner, experience fewer symptoms and possibly even be cured of their disease before it significantly impacts their life. This has benefits for patients but will also improve healthcare for clinicians and payers as well as having global sustainability benefits.


有许多疾病的患者通常都得到同样的治疗,尽管他们的疾病可能有许多不同的原因,并以不同的方式影响他们的健康. For any of these diseases a precision medicine approach could be beneficial. 哪里有现有的治疗方法, precision medicine may offer more effective alternatives by targeting the underlying biology of disease.

Precision medicine could also help people who currently have no treatment options. 一种精确的方法将专注于更具体的患者亚群,因此可能对特定群体产生有效的治疗方法,而不可能找到一种对所有人都有效的治疗方法.


Precision medicine is the result of our deeper scientific understanding of the causes of illness. 新技术支持, 人类基因组测序等进展向澳门葡京赌博游戏表明,慢性疾病的病因是复杂的,同一种疾病可能有许多不同的病因, 每种都需要不同的处理方法. These deeper insights have also made it possible for us to start developing medicines that specifically target the causes of disease.

What is the difference between precision medicine and personalised medicine?

Although the terms are sometimes used interchangeably, many experts use them differently. 澳门葡京赌博游戏使用术语“精准医学”来描述一种治疗方法,这种方法包括根据对疾病潜在原因的科学见解,为特定的患者群体提供最合适的治疗.


采用精准医疗方法有很多好处, 最重要的是,它有可能为患者提供更好的生活质量,并有可能减轻全球医疗保健系统的压力.

Currently, people diagnosed with chronic diseases generally have to live with their effects for the rest of their lives. 通过瞄准疾病的起因, precision medicine has the potential to control the symptoms of disease and, in some cases, could even provide a cure.

Better disease control for patients also means less demand for healthcare services, which are being put under growing pressure due to factors such as increasing population, ageing, 肥胖和COVID-19大流行的后遗症. 连同其他创新, 精准医疗可以减少对广泛医疗的需求, long-term healthcare support, 这意味着更少的预约和更少的住院时间.


Precision medicines are already available for the treatment of some cancers and rare genetic diseases. Currently, precision medicines for chronic diseases are also being researched. Across all areas, precision medicine approaches are being explored for more than 90% of our R&D pipeline.


To develop precision medicines we need an in-depth understanding of the biological changes that cause diseases. This requires extensive scientific insights and advanced technologies, 直到最近才有. For example, 基因测序和基因组编辑有助于确定哪些基因与不同的疾病有关,这使得开发针对这些基因的精准药物成为可能.


No. Many of the first precision medicines have been developed in cancers but the same approach can be applied to many other diseases. Generally, precision medicine is relevant for any disease that can have multiple different causes that all result in a similar set of symptoms. We are currently investigating precision medicine for a wide range of chronic diseases including asthma, 慢性阻塞性肺病,糖尿病以及心血管疾病, renal, 肝脏和免疫疾病.

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1. AstraZeneca Pharmaceuticals. Data on file. Budesonide/formoterol: Annual Rate of Exacerbations Globally (ID:SD-3010-ALL-0017).

2. World Health Organisation. 慢性阻塞性肺病(COPD). Available at:

3. 雷内拉ME,拉撒路JV,拉齐乌V et al. NAFLD命名共识组. A multi-society Delphi consensus statement on new fatty liver disease nomenclature. Hepatology. 2023 Jun 24. doi: 10.1097/HEP.0000000000000520.

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