Peptide and oligonucleotides drugs

Question 1

Advantage and disadvantages

Answer 1

Advantages: High specificity, minimal off-target effects, and mimicry of endogenous molecules.
Challenges: Poor stability, low oral bioavailability, and rapid degradation by enzymes

Question 2

what are the modifications for stability

Answer 2

Peptides: Chemical modifications (e.g., cyclisation, PEGylation) to increase half-life and reduce proteolysis.
Oligonucleotides: Backbone modifications (e.g., phosphorothioates) and conjugation to delivery carriers.

Question 3

describe aso and siRNA drugs

Answer 3

ASO (Antisense Oligonucleotides): Bind mRNA to prevent translation.
siRNA (Small Interfering RNA): Induces mRNA degradation via RNA interference (RNAi).

Question 4

Examples of peptide/oligonucleotide drugs

Answer 4

Peptide: Insulin analogues (e.g., glargine) for diabetes.
Oligonucleotide: Nusinersen (ASO) for spinal muscular atrophy.

Question 5

types if biologic drugs

Answer 5

Monoclonal antibodies (mAbs), vaccines, cytokines, growth factors, and cell therapies.

Question 6

What are monoclonal antibodies

Answer 6

Definition: Antibodies produced from a single B-cell clone targeting specific antigens.
Production: Hybridoma technology or recombinant DNA techniques.

Question 7

types of monoclonal antibodies

Answer 7

Murine: Fully mouse-derived (e.g., muromonab-CD3).
Chimeric: Mouse-human hybrid (e.g., rituximab).
Humanised: Mostly human with mouse antigen-binding regions (e.g., trastuzumab).
Fully Human: Fully human sequence (e.g., adalimumab).

Question 8

why would you choose small molecule or mab projects ?

Answer 8

Small Molecule: Targets intracellular proteins, orally bioavailable.
mAb: High specificity, targets extracellular or surface proteins, requires injection.