Functional genomics
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
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.
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.
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.
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 disease states by manipulating genes in cells or animal models, allowing the effects of new medicines to be tested. Much faster than previous approaches.
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.
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.
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
Specialising in oncology, the AstraZeneca–Cancer Research Horizons Functional Genomics Centre (FGC) is a world-leader in genetic screening, cancer models, CRISPR reagent design and computational approaches to big data processing. We are developing CRISPR technology to better understand the biology of cancer and creating pre-clinical models which are more reflective of human disease.
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: Z4-57937
Date of preparation: September 2023