Brainscape's Knowledge GenomeTM

Browse over 1 million classes created by top students, professors, publishers, and experts.


See full index

LSM4221 > L9: New trends in biotech > Flashcards

L9: New trends in biotech Flashcards

1
Q

what is the biotech evolution?

A

discovery of the following in sequence:
Biotech 0.0: small molecules and recombinant protein
Biotech 1.0: mAbs and Fc fusion
Biotech 2.0: gene transfer and gene therapy
Biotech 3.0: neoantigens and microbiome
Biotech 4.0: engineered gene, extracellular vesicles, digital therapeutics, mitochondria regulation

How well did you know this?
1
Not at all
2
3
4
5
Perfectly
2
Q

what is Kendall square?

A

the heart of biotech, located in cambridge, with many internationally recognised innovation district for research

How well did you know this?
1
Not at all
2
3
4
5
Perfectly
3
Q

state the 2 main elements that makes Kendall square a good ecosystem for biotech

A

due to presence of Lab central and Flagship Ventures

How well did you know this?
1
Not at all
2
3
4
5
Perfectly
4
Q

How does the presence of Lab central and Flagship Ventures make Kendall square a good ecosystem?

A

Lab central: an incubator for start up companies. people from MIT are innovative and retn space to start proj once approved. can propose projects to investors eg. flagship ventures and prove that pdt is able to be commercialised [a form of validation]
- big companies also move into lab central to look for talents and ideas

Flagship ventures: investor of lab central

How well did you know this?
1
Not at all
2
3
4
5
Perfectly
5
Q

what is the trending thing now?

A

developing gene therapy

How well did you know this?
1
Not at all
2
3
4
5
Perfectly
6
Q

how does gene therapy work? [4]

A
  1. gene replacement: replace non-working mutant gene w a healthy one, eg. over-express healthy gene is disease is loss of function
  2. gene silencing: inactivate mutant gene that is toxic for the cells
  3. gene addition: over-expression of a foreign gene to impact disease state
  4. gene editing: changes the genome by permanent manipulation
How well did you know this?
1
Not at all
2
3
4
5
Perfectly
7
Q

how are gene delivered?

A
  1. viral vectors: retroviruses, adenoviruses, adeno-associated viruses–> gene inserted into viral genome and then inserted into our genome when virus infect us
  2. non-viral vectors: liposomes, naked dna
How well did you know this?
1
Not at all
2
3
4
5
Perfectly
8
Q

problem w using viral vectors for gene therapies [2]

A
  1. retroviruses integrate viral genome randomly into human cells
  2. viral genome have promoters that contain other unwanted gene eg. oncogene Jesse Gelsinger
How well did you know this?
1
Not at all
2
3
4
5
Perfectly
9
Q

unique benefits of gene therapy [5]

A
  1. solve unmet needs: esp for treating rare diseases w few/no treatment options
  2. current approved therapies have high efficacy
  3. targets cause of disease
  4. reduce/eliminate need for other costly treatment eg bone marrow transplant w high risk of infection
  5. less expensive than doing screening to find specific small molecule to treat disease
How well did you know this?
1
Not at all
2
3
4
5
Perfectly
10
Q

why is developing gene therapy “better” than developing small molecule drug?

A

small molecule drug:
- takes a few years to screen for a compound w therapeutic effect
- more time needed to know how compound acts on body, mechanism [TAKES TOO LONG]

gene therapy: skips the screening steps, as we skip straight to gene associated with disease

How well did you know this?
1
Not at all
2
3
4
5
Perfectly
11
Q

most of the gene therapies in development are in which phase?

A

phase I/II

How well did you know this?
1
Not at all
2
3
4
5
Perfectly
12
Q

what is the Orphan Drug Act?

A

an incentive to develop drugs for rare diseases, using eg. tax credits, market exclusivity, application, reduced statistic requirement

How well did you know this?
1
Not at all
2
3
4
5
Perfectly
13
Q

current challenges of gene therapy [3]

A
  1. restoration of missing gene not alws sufficient to cure a disease
  2. there are limited popn of patients w rare disease for clinical trials
  3. challenging delivery of RNA and DNA
How well did you know this?
1
Not at all
2
3
4
5
Perfectly
14
Q

which has less side effects? using nanoparticles or viral vectors to deliver RNA/DNA?

A

nanoparticles, tho it can still elicit immune response

How well did you know this?
1
Not at all
2
3
4
5
Perfectly
15
Q

can nanoparticles be injected into systemic circulation?

A

no, can only inject into muscles

How well did you know this?
1
Not at all
2
3
4
5
Perfectly
16
Q

how to stabilise mrna vector? side effect of this meethod?

A

use polyethylene glycol. can cause anaphylactic shock

How well did you know this?
1
Not at all
2
3
4
5
Perfectly
17
Q

limitations of using viral vectors [4]

A
  1. immunogenicity
  2. limited payload capacity
  3. risk of insertional mutagenesis
  4. manufacturing challenge
18
Q

limitations of using lipid nanoparticles [3]

A
  1. limited biodistribution
    2.toxicity at high doses
  2. low efficiency of endosomal escape
19
Q

how does extracellular vesicles solve existing limitations in nucleic acid delivery? [5]

A
  • are natural carriers of RNAs [siRNAs, mRNAs]
  • offers great biocompatibility: robust uptake by many cell types, low toxicity and immunogenicity
  • may avoid phagocytosis and multi-drug resistance
20
Q

what cells are good sources of EVs?

A

red blood cells

21
Q

which is better, using EVs from cell lines or RBCs and why?

A

RBCs is better.

Problem with EVs from cell lines:
-scale up and CMC, require MASSIVE cell culture to achieve single human dose
- safety: oncogenic components from cell lines may cause oncogenesis [her2,cmyc] and from genetic manipulation of cells

RBcs:
-no risk of oncogenesis and genetic material transfer
- sustainable since we have alot and is readily opbtained
- scalable: formation of RBC EVs can be accelerated in economical fashion

22
Q

what does REGENT stand for?

A

red cell EV gene therapy

23
Q

why is REGENT an efficient delivery system? [3]

A
  1. large payload capacity: can put large amts of genes into EVs
  2. high copy number
  3. non-immunogenic
24
Q

what are the manufacturing advantages of REGENT? [3]

A
  1. simple and fast loading method
  2. propriety surface modification method
    -3. dont require expensive cell culture or genetic manipulation of cells
25
Q

elab on the of-the-shelf availability of REGENT [3]

A
  1. allogeneic O-ve blood donor sourced
  2. can do repeated freeze-thaw cycles without lysis
  3. use lyophilization [free drying] for long term storage at ambient temp
26
Q

elab on the biodistribution of using REGENT

A
  1. can be distributed to liver/hepatic**, spleen, bone marrow and lungs
  2. can have targeted delivery to specific organs/tissues
27
Q

what are the key elements of starting a biotech company? [8]
Hint: IIL BIITS

A
  1. innovation: novel platform technology
  2. IP: proprietary and protected intellectual property
  3. leader: strong CEO
  4. Business: a solid business plan
  5. investment: solid financial backing by venture capital
  6. Infrastructure: incubator lab space w all the necessary equipment, helpful govt and regulatory environ.
  7. team: talented and experienced R&D team
  8. SAB: top scientists in the scientific advisory board
28
Q
  1. innovation:
A

novel platform technology

29
Q
  1. IP:
A

proprietary and protected intellectual property

30
Q
  1. leader:
A

strong CEO

31
Q
  1. Business:
A

solid business plan

32
Q
  1. investment:
A

solid financial backing by venture capital

33
Q
  1. Infrastructure:
A

incubator lab w necessary equipment, helpful govt and regulatory environ

34
Q
  1. team:
A

talented and experienced r&d team

35
Q
  1. SAB:
A

top scientists in the scientific advisory board

36
Q

first group of employees get shares in start-up companies. T/F?

A

quite true

37
Q

start-up jobs are more risky. T/F?

A

both. chance of failing is high for company but skills are transferable and start-ups can jump from one to another

38
Q

summary of the biotech evolution

A
  1. started with DNA cloning and recombinant proteins
  2. then to therapeutic antibodies
  3. new trend: cell and gene therapy
39
Q

why is gene therapy getting more investments?

A

due to Orphan drug act that promotes companies to research on treatment for rare diseases

40
Q

why is REGENT a good platform for expanding RBCs for gene therapy? [4 brief points]

A
  1. Biodistribution [extra hepatic distribution + specific delivery to tissues] [2]
  2. Availability: on the shelf availability to many people [3]
  3. Delivery is efficient [3]
  4. Manufacturing advantages [3]

LSM4221 (13 decks)

Brainscape helps you reach your goals faster, through stronger study habits.
© 2024 Bold Learning Solutions. Terms and Conditions