How do we decide whether to start a drug discovery project Flashcards
1
Q
What is the 5R framework?
A
- Right target
- Right tissue – molecule gets to right place where needed
- Right safety - minimal side effects
- Right patient
- Right commercial – future viability/ what’s already on the market
2
Q
How do we come up with the right target?
A
- Functional genomics
- Multi Omics
- Artificial intelligence and machine learning
3
Q
What is functional genomics
A
• Investigates link between information contained in our DNA and the functional effect of this information
4
Q
What is crispr9
A
- A way of editing the genome
- Future therapeutic in its own right
- Can identify genes responsible for normal biological and disease processes
- Cas9 – enzyme that can cut DNA
5
Q
What is multi omics
A
- Data sets of difference omic groups are combined during analysis
- Uses AI to make models
- Identify new drug targets – biomarkers or toxicity
- Can be used for drug repurposing
- Personalised medicine approach e
6
Q
What are omic groups
A
• Branches of science e.g., genomics, proteomics (set of proteins), metabolomics
7
Q
What technology does multi omics use?
A
DNA microarray
• Next generation sequencing for genomics
• Mass spec to measure proteins, lipids and metabolites
8
Q
What is artificial intelligence and machine learning?
A
- AI can help us understand and use information to select right targets
- Creates knowledge graphs – networks of scientific facts
- Use this data to make disease hypothesis
9
Q
What are next generation therapeutics?
A
- Small molecules e.g., PROTACs
- Antibody therapeutics e.g., monoclonal antibodies
- Cell based therapeutics
- Peptide or protein therapeutics
- Nucleotide-based therapeutics e.g. mRNA
