The field of pharmaceutical biotechnology is characterized by breathtaking innovation, offering unprecedented opportunities to treat and even cure complex diseases. However, this rapid progress is matched by significant scientific, manufacturing, and regulatory challenges. For PharmD students, understanding this landscape of challenges and opportunities is essential for appreciating the drug development lifecycle, the value and risks of novel therapies, and the future direction of pharmacy practice.
1. A significant challenge for the repeated administration of an in vivo gene therapy using a viral vector like adenovirus is:
- The vector’s inability to carry a genetic payload.
- The development of a host immune response against the vector, limiting its effectiveness on subsequent doses.
- The extremely low cost of production.
- The lack of target diseases for this approach.
Answer: The development of a host immune response against the vector, limiting its effectiveness on subsequent doses.
2. The opportunity to treat diseases caused by a single faulty gene with a one-time, potentially curative treatment is a primary driver for which technology?
- Monoclonal antibody therapy
- Gene editing and gene therapy
- Small molecule drug development
- Herbal supplement research
Answer: Gene editing and gene therapy
3. A major manufacturing challenge for autologous cell therapies (like CAR-T) is the:
- Simplicity and low cost of the process
- Ability to make one large batch to treat all patients
- “Scale-out” problem, where each batch is a custom product for a single patient
- Long-term stability of the product at room temperature
Answer: “Scale-out” problem, where each batch is a custom product for a single patient
4. The rapid development of mRNA vaccines during the COVID-19 pandemic highlighted a major opportunity for:
- Using this platform technology for rapid response to future pandemics
- Creating vaccines that are stable at all temperatures
- Developing vaccines that can be administered orally
- A low-cost alternative to all other vaccine types
Answer: Using this platform technology for rapid response to future pandemics
5. What is considered the greatest challenge for the clinical success of RNA-based therapeutics like siRNA and ASOs?
- Their extreme stability in the bloodstream
- A lack of specific molecular targets
- Efficient and targeted delivery to the desired cells or tissues
- Their simple and inexpensive manufacturing process
Answer: Efficient and targeted delivery to the desired cells or tissues
6. The “cold chain” represents a significant logistical challenge for which class of drugs?
- Small-molecule oral tablets
- Biologics like proteins and monoclonal antibodies
- Topical creams and ointments
- Saline solutions
Answer: Biologics like proteins and monoclonal antibodies
7. A key opportunity presented by induced pluripotent stem cells (iPSCs) is the ability to:
- Create patient-specific cells for disease modeling and drug screening
- Avoid all ethical considerations in research
- Directly create fully formed organs for transplant immediately
- Use them without risk of tumor formation
Answer: Create patient-specific cells for disease modeling and drug screening
8. The high cost of development for biologics is a major challenge, partly due to the complexity of:
- Upstream (cell culture) and downstream (purification) processing
- Writing the prescription in the pharmacy
- Storing the raw materials, which are inexpensive
- The simple regulatory approval process
Answer: Upstream (cell culture) and downstream (purification) processing
9. The risk of “off-target effects” is a primary safety challenge for which of the following technologies?
- Gene editing with CRISPR-Cas9
- Monoclonal antibody therapy
- Subunit vaccine administration
- Small molecule drug absorption
Answer: Gene editing with CRISPR-Cas9
10. RNA therapeutics offer the opportunity to target molecules that are considered “undruggable” by traditional small molecules, such as:
- Cell surface receptors
- Specific mRNA transcripts
- Extracellular enzymes
- Ion channels
Answer: Specific mRNA transcripts
11. A significant regulatory challenge for biosimilars is:
- Proving that they are chemically identical to the reference small molecule
- Demonstrating a high degree of similarity to the reference biologic with no clinically meaningful differences
- The very short patent life of the original biologic
- The lack of any FDA guidance on their development
Answer: Demonstrating a high degree of similarity to the reference biologic with no clinically meaningful differences
12. The use of exosomes as a “liquid biopsy” presents an opportunity for:
- A new method of oral drug delivery
- Early disease diagnosis and monitoring through biomarkers found in bodily fluids
- A cheaper way to manufacture monoclonal antibodies
- A type of stem cell therapy
Answer: Early disease diagnosis and monitoring through biomarkers found in bodily fluids
13. What is the primary ethical challenge associated with human germline gene editing?
- The edits would be heritable, affecting all future generations
- The technology is not effective
- The cost is prohibitive for most patients
- It can only be performed on adult somatic cells
Answer: The edits would be heritable, affecting all future generations
14. An opportunity for improving protein therapeutics is PEGylation, a process that can:
- Increase the protein’s immunogenicity
- Decrease the protein’s circulating half-life
- Increase the protein’s circulating half-life, allowing for less frequent dosing
- Make the protein suitable for oral administration
Answer: Increase the protein’s circulating half-life, allowing for less frequent dosing
15. A major challenge in developing therapies using pluripotent stem cells (ESCs/iPSCs) is the risk of:
- The cells failing to self-renew
- The cells differentiating too slowly
- Teratoma formation if undifferentiated cells remain in the final product
- The cells being too small to be effective
Answer: Teratoma formation if undifferentiated cells remain in the final product
16. The concept of personalized medicine is a major opportunity that relies on:
- Treating all patients with the same drug and dose
- Using biomarkers and pharmacogenomics to tailor treatment to individual patients
- Focusing only on lifestyle and diet modifications
- Using the least expensive drug available, regardless of efficacy
Answer: Using biomarkers and pharmacogenomics to tailor treatment to individual patients
17. A challenge for gene pharming (producing drugs in transgenic animals/plants) is:
- The rapid speed of development from concept to product
- Public perception and complex regulatory oversight
- The simplicity of the purification process
- The ability to produce only small molecule drugs
Answer: Public perception and complex regulatory oversight
18. The development of antibody-drug conjugates (ADCs) represents an opportunity to:
- Decrease the specificity of cancer therapy
- Increase the systemic toxicity of chemotherapy
- Use antibodies to deliver a potent cytotoxic payload directly to cancer cells
- Replace the need for all monoclonal antibodies
Answer: Use antibodies to deliver a potent cytotoxic payload directly to cancer cells
19. A key challenge in the “valley of death” in drug development is:
- Securing funding to move a promising discovery from basic research into clinical trials
- The final marketing of an FDA-approved drug
- The initial discovery of a new biological target
- Performing post-market surveillance
Answer: Securing funding to move a promising discovery from basic research into clinical trials
20. A challenge in using ZFNs and TALENs for gene editing is that:
- They are easier to design than CRISPR systems
- They require complex protein engineering for each new DNA target
- They do not use a nuclease to cut DNA
- They have no off-target effects
Answer: They require complex protein engineering for each new DNA target
21. The opportunity for “in-situ protein replacement” is best demonstrated by which technology?
- Monoclonal antibody infusion
- mRNA therapeutics that provide a template for protein production
- Antisense oligonucleotides
- Stem cell transplantation
Answer: mRNA therapeutics that provide a template for protein production
22. A major scientific challenge for all biologic drugs is immunogenicity, which is:
- The ability of the drug to suppress the immune system
- The tendency of the drug to provoke an unwanted immune response
- The measure of the drug’s therapeutic effect
- The drug’s rate of clearance from the body
Answer: The tendency of the drug to provoke an unwanted immune response
23. The high cost of many novel biotechnologies presents a major societal challenge related to:
- Patient access and health equity
- A lack of innovation
- The ease of manufacturing
- The short duration of patents
Answer: Patient access and health equity
24. The development of oral formulations for protein drugs like insulin is a major opportunity, but is challenged by:
- The protein’s stability in the GI tract and poor absorption
- The protein’s high stability in acidic environments
- The small size of most protein drugs
- The preference of all patients for injections
Answer: The protein’s stability in the GI tract and poor absorption
25. A challenge for the isolation of therapeutic exosomes is:
- Separating them from other extracellular vesicles and protein aggregates
- Their large size, which simplifies filtration
- The fact that cells produce only one type of exosome
- Their extreme durability under harsh chemical conditions
Answer: Separating them from other extracellular vesicles and protein aggregates
26. The opportunity to use CRISPR for diagnostics (e.g., SHERLOCK) is based on its ability to:
- Precisely recognize specific nucleic acid sequences
- Cut proteins instead of DNA
- Behave as an antibody
- Generate a strong light signal on its own
Answer: Precisely recognize specific nucleic acid sequences
27. A key challenge for the pharmacist managing a patient on a novel cell therapy is:
- The simple and well-known side effect profile
- The lack of special storage and handling requirements
- Understanding complex protocols, managing unique toxicities, and coordinating care
- The ability to substitute it with a generic small molecule
Answer: Understanding complex protocols, managing unique toxicities, and coordinating care
28. An opportunity presented by CAR-T cell therapy is:
- A new, low-cost oral treatment for leukemia
- A potentially curative “living drug” for certain hematologic cancers
- A therapy with no significant side effects
- A treatment that does not require patient-specific manufacturing
Answer: A potentially curative “living drug” for certain hematologic cancers
29. The need for long-term follow-up studies for gene therapies is a challenge because:
- The effects are known to be temporary
- Potential long-term risks, such as secondary malignancies, must be monitored
- The FDA does not require any follow-up after approval
- Patients are always cured and require no further monitoring
Answer: Potential long-term risks, such as secondary malignancies, must be monitored
30. The use of artificial intelligence and machine learning in biotechnology presents an opportunity to:
- Replace all laboratory researchers
- Accelerate drug discovery by predicting protein structures and identifying new drug targets
- Eliminate the need for clinical trials
- Make the drug manufacturing process more expensive
Answer: Accelerate drug discovery by predicting protein structures and identifying new drug targets
31. A challenge for ex vivo gene therapies is the need to successfully ________ the edited cells back into the patient.
- digest
- engraft
- excrete
- radiate
Answer: engraft
32. The opportunity to create “bio-betters” refers to:
- Developing an exact copy of an existing biologic drug
- Engineering a new version of an existing biologic with improved properties
- A generic version of a small molecule drug
- A lower-cost, lower-quality version of a biologic
Answer: Engineering a new version of an existing biologic with improved properties
33. The complexity of intellectual property rights and patent disputes is a major business challenge in which area?
- The use of generic aspirin
- The field of gene editing, particularly CRISPR
- The compounding of simple suspensions
- The sale of over-the-counter vitamins
Answer: The field of gene editing, particularly CRISPR
34. The opportunity for gene silencing with RNAi is most promising for diseases caused by:
- The lack of a functional protein
- The production of a toxic, disease-causing protein
- A bacterial infection
- A nutritional deficiency
Answer: The production of a toxic, disease-causing protein
35. A challenge for the entire field of biopharmaceuticals is ensuring a well-trained workforce capable of handling:
- Complex manufacturing, quality control, and clinical management
- Simple chemical synthesis only
- Retail sales and marketing exclusively
- The same tasks required for small molecule drugs
Answer: Complex manufacturing, quality control, and clinical management
36. The opportunity for exosomes to be used as a drug delivery vehicle to the brain is based on their potential to:
- Be actively transported across the blood-brain barrier
- Easily diffuse through all biological membranes
- Disassemble before reaching the brain
- Bind to and neutralize circulating toxins
Answer: Be actively transported across the blood-brain barrier
37. A significant challenge in using viral vectors is their limited “packaging capacity,” which refers to:
- The maximum size of the therapeutic gene they can carry
- The type of box they are shipped in
- The number of viruses that can fit in a vial
- The speed at which they can be manufactured
Answer: The maximum size of the therapeutic gene they can carry
38. The opportunity to develop edible vaccines in transgenic plants faces the challenge of:
- Ensuring consistent dosing and stability through the GI tract
- The high cost of growing plants
- The inability of plants to produce proteins
- The risk of causing animal-based diseases
Answer: Ensuring consistent dosing and stability through the GI tract
39. A major challenge for the clinical adoption of any new biotechnology is:
- Generating robust safety and efficacy data from well-designed clinical trials
- Keeping the technology a secret from the public
- Avoiding all interaction with regulatory bodies like the FDA
- Ensuring the treatment is as difficult to administer as possible
Answer: Generating robust safety and efficacy data from well-designed clinical trials
40. The development of high-fidelity Cas9 enzymes is a strategy to overcome which challenge?
- The enzyme’s inability to cut DNA
- Off-target effects of gene editing
- The need for a guide RNA
- The high cost of the enzyme
Answer: Off-target effects of gene editing
41. An opportunity for personalized cancer therapy involves using a patient’s own tumor genetics to:
- Select a targeted therapy most likely to be effective
- Choose a generic antibiotic
- Prescribe the highest possible dose of chemotherapy
- Administer a one-size-fits-all treatment protocol
Answer: Select a targeted therapy most likely to be effective
42. A challenge for regenerative medicine using stem cells is controlling their:
- Self-renewal and differentiation pathways after transplantation
- Ability to be stored at room temperature
- Color and shape
- Inability to divide
Answer: Self-renewal and differentiation pathways after transplantation
43. The success of antibody-drug conjugates (ADCs) has created an opportunity to revisit chemotherapy agents that were:
- Too potent and had too much systemic toxicity to be used on their own
- Not potent enough to be effective
- Ineffective against all types of cancer
- Unable to be synthesized
Answer: Too potent and had too much systemic toxicity to be used on their own
44. A major challenge for any therapy requiring ex vivo cell manipulation is the:
- Risk of contamination during the cell culture process
- Simplicity and speed of the procedure
- Low cost associated with personalized manufacturing
- Lack of need for specialized facilities
Answer: Risk of contamination during the cell culture process
45. The opportunity for biotechnology to address rare, or “orphan,” diseases is significant, but is challenged by:
- The large number of patients available for clinical trials
- The small patient population, which makes it difficult to recoup development costs
- The simplicity of the underlying genetics of these diseases
- A lack of interest from patient advocacy groups
Answer: The small patient population, which makes it difficult to recoup development costs
46. The ability to create “knockout” animal models using gene editing provides an opportunity to:
- Study the function of a specific gene by observing the effect of its absence
- Create a healthier and more robust animal
- Ensure the animal will live forever
- Test new types of food supplements
Answer: Study the function of a specific gene by observing the effect of its absence
47. A challenge in developing RNA aptamers is ensuring their:
- Lack of specificity for the target
- Stability against nuclease degradation in the body
- Inability to fold into a 3D structure
- High level of immunogenicity
Answer: Stability against nuclease degradation in the body
48. An opportunity for pharmacists in the age of biotechnology is to:
- Transition away from patient care roles
- Specialize in the management of complex biologic and cell/gene therapies
- Focus solely on dispensing small molecule drugs
- Ignore all new drug developments
Answer: Specialize in the management of complex biologic and cell/gene therapies
49. The development of “bio-inks” and 3D bioprinting using stem cells presents a long-term opportunity for:
- Creating custom-printed medications
- Fabricating human tissues and organs for transplantation
- A new type of diagnostic imaging
- A novel vaccine delivery system
Answer: Fabricating human tissues and organs for transplantation
50. The overarching challenge and opportunity for the entire biotechnology industry is to:
- Maintain the status quo and avoid innovation
- Balance groundbreaking scientific potential with the practical realities of safety, regulation, and cost
- Focus only on developing the most expensive treatments possible
- Keep clinical trial data secret from regulators and the public
Answer: Balance groundbreaking scientific potential with the practical realities of safety, regulation, and cost
I am a Registered Pharmacist under the Pharmacy Act, 1948, and the founder of PharmacyFreak.com. I hold a Bachelor of Pharmacy degree from Rungta College of Pharmaceutical Science and Research. With a strong academic foundation and practical knowledge, I am committed to providing accurate, easy-to-understand content to support pharmacy students and professionals. My aim is to make complex pharmaceutical concepts accessible and useful for real-world application.
Mail- Sachin@pharmacyfreak.com