FINAL: L1

Question 1

Biotherapeutics

Answer 1

Large, “living” drugs

Question 2

Biological products have ____ specificities against the target

Answer 2

Molecular

Question 3

Bioterapeutics activity is driven by their

Answer 3

Structure and their interaction with cellular components
-E.g., a cell can sense the microenvironment; a gene can alter the phenotype of a cell

Question 4

Where do Biptherapeutics go

Answer 4

DNA therapeutics
Protein Therapeutics
RNA therapeutics
Cell-based therapies

Question 5

CAR-T Therapies

Answer 5

Abecma (2021)
Celgene

Breyanzi (2021) J
Juno Therapeutics

Carvykti (2022)
Janssen Biotech inc.

Kymirah (2017)
Novartis Pharm Corp

Tecartus (2020)
Kite Pharma Inc

Yescarta (2017)
Kite Pharma Inc

Question 6

Cell therapies

Question 7

A ____ therapy is a gene therapy when it delivers or is engineered with a gene

Answer 7

Cell based therapy

Question 8

Cell based therapies vs Gene therapies

Answer 8

Cell based:
-Non-modified cell therapy
-Stem cell therapy

Gene Therapies
-Virus-delivered genes
-Gene-edited cells

FDA has approved both cell-based gene therapies and non-gene based cell therapies

Question 9

Cell therapies can contain genes and antibodies

Answer 9

Gene therapy:
DNA, viral vector

Cell-based therapy:
Expressed Gene
Immune cell

Antibody Drug
-Antibody drug conjugate

Question 10

Classes of cell based therapies:

Answer 10

** Stem cells and Stem Cell-Derived products**
-Do NOT involve immune cells
-E.g. hematopoietic, mesenchymal, embryonic, umbilical cord blood, etc

Immunotherapies
-Involve immune cells
-Cancer vaccines and Cell-based immune therapies
-E.g. dendritic cells (typically vaccines), genetically-modified lymphocytes, activated lymphocytes (e.g. TIL, NK, T), cancer cells chemically modified or unmodified

Immunotherapies include native and cell-based gene therapies (genetically-engineered lymphocytes)