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CRISPR gene editing
This approach is underpinned by our ability to rapidly gain a deeper understanding of disease biology and identify a wealth of potential drug targets that may hold the keys for unlocking new treatments and potential cures. We are using a multi-pronged approach – investing in genomics, data science and AI, next-generation sequencing and <anchor>CRISPR gene editing</anchor> – to identify, interrogate, and validate new targets in a way not previously thought possible. Each new insight and every new piece of information brings us closer to creating better, more advanced and more effective treatments for patients.
Functional genomics
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# Functional genomics

Understanding diseases and enabling drug discovery through CRISPR gene editing

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## What is functional genomics?

### Functional genomics is a rapidly advancing drug discovery method that can be used to explore links between our genes and their effects. Using functional genomics, we can identify and investigate genes and biological processes that are associated with diseases and which can be explored as potential drug targets.

Genome editing is a key tool in functional genomics, making it possible to delete or change genes in cells to understand their roles in disease. One of the most well-known genome editing tools is CRISPR.

## Genome editing technologies in functional genomics

Find out about gene editing technology and how CRISPR is adding to our understanding of disease.

A number of technologies are available to study functional genomics. But by far the most effective and versatile is the revolutionary gene editing technology CRISPR/Cas9 – or CRISPR (Clustered Regularly Interspaced Short Palindromic Repeat). In 2015 it was named the breakthrough of the year by Science magazine and has rapidly become a widespread and much valued research tool.

CRISPR represents a breakthrough approach to editing genes and many variants have been developed. In its simplest application, CRISPR acts as molecular scissors that can be used to precisely cut and modify a DNA sequence of interest. Accurate, programmable and adaptable, this technology has found widespread application across several areas of biological and biopharmaceutical research.

We were one of the first pharmaceutical companies to invest in CRISPR, setting up dedicated research groups in Gothenburg and Cambridge in 2014. The ability to ‘edit’ genes using CRISPR may be one of the most significant discoveries in the history of biology. It not only has potential as a tool to study and understand disease in functional genomics, but in the future even has potential to treat disease with a genetic cause.

## Recent updates in functional genomics

## How does functional genomics support drug discovery?

### Linking genes to disease

The primary reason that candidate drugs fail in clinical trials is because they do not have a significant effect on the course of disease.

Selecting the right target is therefore the single most important decision we make in the drug discovery process.

### Finding the right targets

Functional genomics helps us to identify the right targets for drug discovery, uncovering genes and biological processes that can be targeted to help slow, stop or reverse disease.

By using functional genomics to guide drug discovery we can deliver precision medicines that target the underlying causes of disease.

### Contributing to clinical success

We are using functional genomics to guide our drug discovery process, improving our chances of success in clinical trials. Ultimately this can help to deliver more life-changing medicines sooner.

## How is functional genomics being used?

We are exploring several ways that functional genomics can be used to support drug discovery. Increasingly, we are able to perform large-scale CRISPR screens that integrate data science and artificial intelligence (AI) to help uncover meaningful patterns in the data.

![ ](/content/dam/az/wscd/tile/CRISPR_1-2 RGB Mulb.Gold_900x500.jpg/jcr:content/renditions/cq5dam.web.620.CRISPR_1-2 RGB Mulb.Gold_900x500.jpg)

## Gene knock-outs

Examining the effect of removing a gene from the genome. Widely used to identify genes that when deleted lead to resistance to cancer medicines.

 

## Disease models

Simulating human disease states by manipulating genes in cells and animal models, allowing the effects of potential new medicines to be tested. Much faster than previous approaches.

![ ](/content/dam/az/wscd/tile/Analytics_2-3 RGB Navy-Green_900x501.jpg/jcr:content/renditions/cq5dam.web.620.Analytics_2-3 RGB Navy-Green_900x501.jpg)

## High-throughput screening

Combining biology and computing to analyse large amounts of CRISPR data to understand ‘big picture’ links between genes and disease using machine learning and AI.

## Can you help us find more life-changing medicines? Apply to join the team.

## Collaborating to discover new therapies with functional genomics

### Human Functional Genomics Initiative

Together with the Medical Research Council and the Milner Therapeutics Institute, we are creating a functional genomics laboratory, capable of large-scale CRISPR screening. The facility supports our BioPharmaceuticals ambition of transforming care for people with chronic diseases.

This unique collaboration enables open sharing of resources and expertise across academia and industry, and plays a focal role in the UK-wide Human Functional Genomics Initiative. The lab supports discovery research in diseases such as asthma, cardiovascular, metabolic and inflammatory diseases.

### Functional Genomics Centre with Cancer Research Horizons

Established in 2019, the Functional Genomics Centre (FGC) is a world-leading centre of excellence in genetic screens, cancer models, CRISPR vector design and computational approaches to big data. Specialising in oncology, the goal is to identify novel targets and resistance mechanisms to help accelerate the discovery of new cancer medicines.

The FGC is a unique partnership between Cancer Research UK’s (CRUK) drug discovery engine, Cancer Research Horizons and AstraZeneca. This industry-academia partnership, which aims to democratise access to functional genomics across the CRUK research community, enables AstraZeneca and Cancer Research Horizons to work together to solve some of the most important problems in cancer today.

### Wellcome Sanger Institute

Since 2015, we have collaborated with the Wellcome Sanger Institute on CRISPR technology. This collaboration was extended in 2018, giving us access to its leading libraries of gRNA for silencing or activating every gene in the genome via CRISPR.

We can do more faster by working together. Our functional genomics partnerships are revealing more insights into disease biology and allowing shared access to the latest technologies and leading expertise.

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Veeva ID: Z4-65666

Date of preparation: June 2024

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Veeva ID: Z4-46798

Date of next review: August 2024

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