Wk.9 L2 - Biologics

Question 1

LO

Answer 1

• Describe the differences between biologics and conventional small-molecule drugs.
• Describe the different types of biologics.
• Describe the mechanisms of action for different biologics.
• Describe the challenges associated with different biologics.
• Correlate drug suffixes with their respective drug classes and therapeutic uses

Question 2

biologics

Answer 2

Range of products:
- Vaccines
- Blood
- Cells/ tissue

Can be composed of:
- sugars
- proteins
- Nucleic acids
- Combinations w/ living cells

Isolated from natural sources (animal/ human)

Question 3

Blood

Answer 3

• Blood infusion
• Considered to be a biologic as it cannot be chemically synthesised
• Components of the blood

Question 4

Insulin

Answer 4

• Biologic example
• Treats #1 Diabetes

Question 5

Small molecules Vs biologics

Answer 5

• Biologics are much larger, mroe complex and come from living cells
• Administration routes differ; biologics are injected or IV, rather than tablets/ liquid injested/ sprayed
• Biologics are more sensitive to storage and handling conditions

Question 6

Biologics categories

Answer 6

• Nucleic acid therapeutics
• Protein-based therapeutics
• Cell-based therapeutics

Question 7

Nucleic acid therapeutics

Answer 7

Use DNA/ RNA to regulate gene expression

Antisense oligonucleotides (ASO)
- Degradation, translation inhibition, splicing

RNA interference (RNAi)
- Degradation

Messenger RNA (mRNA)
- Delivering synthetix mRNA

Question 8

Nucleic acid therapeutics challenges

Answer 8

Too large and -ve charged to entre cell alone
Injected into site/ region
Lipid nanoparticles help it get into cell

Question 9

Protein-based therapeutics

Answer 9

Peptides
- Glucagon-like peptide-1 (GLP1) receptor agonists
- Increases insulin in pancreas
- Decreases appetite in brain & blood psi in vessels
- Decrease gastic emprying in stomach
- Used to manage #2 Diabetes & obesity

Enzymes (replacing missing or defective enzymes)

Antibodies (covered in another lecture)

Question 10

Protein-based therapeutics challenges

Answer 10

• Poor ability to be absorbed properly
• Can be degraded
• Parid renal clearance
• Cannot cross membrane alone
• Intracellular targetting

Question 11

Cell-based therapeutics

Answer 11

CAR-T cells
Stem cells
Microbiome

Question 12

Autologous CAR-T cell therapy

Answer 12

Using pateints Tcells & genetically modifying to target specofoc tumour antigens
- Kill cancer efficiently

Question 13

Autologous CAR-T cell therapy Challenges

Answer 13

Labour intensive and time consuming
To face the challenge, allosteric CAR-T cells are being produced for “off-the-shelf”
- But has own challenges (immune rejection)

Question 14

Allogeneic stem cell transplantation

Answer 14

Harvest healthy donor stem cells
Patients immune system is suppressed via chemotherapy, so the new stem cells arent rejected
Stem cells are injected into patient and travel to bone marrow to create new healthy blood cells

Question 15

Allogeneic stem cell transplantation challenges

Answer 15

Donor matching
Immune rejection

Question 16

Faecal Microbiota Transplant

Answer 16

Antobiotics kill the gt microbiome, infection then starts to grow in gut
Take faeceal matter from donor and transplant into patient via rectal route in liquid form
Gives patient a dose of healthy microbiota, hopefully colonising gut to fight infection

Question 17

Faecal Microbiota Transplant challenges

Answer 17

• Donor screening
• Infection risks
• Standardising manufacture and storage