Functional genomics

Understanding diseases and enabling drug discovery through CRISPR gene editing


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?





By targeting every single gene in the genome and understanding the networks in which they function, hand-in-hand with novel treatment approaches such as antisense oligonucleotides, we can expand the therapeutic world that is available to us.

Steve Rees SVP, Discovery Sciences, BioPharmaceuticals R&D, AstraZeneca



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.



The key to maximising the chances of success in target identification lies in the pairing of the latest genome editing technologies with bioinformatics and AI to efficiently analyse the data generated from screenings.

Davide Gianni Senior Director, Functional Genomics, Discovery Sciences, R&D, AstraZeneca



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


AstraZeneca supports me to develop my skills with some of the latest technologies, and to work closely with a wide range of leading experts in genome editing.





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