11 - drug discovery 3 Flashcards
What is the MOA of antisense oligonucleotides (ASO)?
Blocks the mRNA directly to reduce amount of protein made
Example of an ASO.
Patisiran (Onpattro)
What is Patisiran (Onpattro)?
RNAi for hereditary transthyretin-mediated amyloidosis
What is the MOA of mRNA medicine?
Introduces exogenous mRNA into body, so cells can make proteins based on introduced mRNA
Example of mRNA medicine.
COVID-19 vaccine
What is required in a mRNA medicine?
- mRNA sequence
- “Right” coating
- RNA sequence
What is the MOA of monoclonal antibody and antibody-drug conjugate?
- A type of protein that specifically binds to its target to block the function of that target protein
- Or help recognize a specific group of cells that express that target protein
Example of monoclonal antibody.
PD1 and PD-L1
How does gene therapy with adeno-associated virus (AAV) work?
- Transgene is packaged into AAV vectors
- Through an infusion, AAVs carrying the transgene targets the liver
- AAV vectors deliver transgene to nucleus of liver cells to enable production of therapeutic protein
What is an example of a drug that is a gene therapy w adeno-associated virus?
Zolgensma
What is Zolgensma?
A biologic drug consisting of adeno-associated virus (AAV9) capsids that contain SMN1 transgene along w synthetic promoters to tx spinal muscular atrophy (SMA)
What is SMA?
A neuromuscular disorder caused by a mutation in SMN1 gene, which leads to decrease in SMN protein
What are SMN proteins?
A protein necessary for survival of motor neurons
What is CRISPR/Cas9?
- A DNA editing technique
- Works like a biological version of word-processing program’s “find and replace” function
What does CRISPR stand for?
Cluster regularly interspaced short palindromic repeats
