Biopharmaceuticals and gene therapy Flashcards
What is a biopharmaceutical?
A biopharmaceutical is a complex drug derived from biological sources. It is a biological molecule used as a pharmaceutical, which can either be a protein or an oligonucleotide.
What is the difference between first and second-generation biopharmaceuticals?
First-generation biopharmaceuticals have no structural changes made to the native molecule, whereas second-generation biopharmaceuticals involve modifications to the structure of the molecule.
What is an oligonucleotide?
An oligonucleotide is a short DNA or RNA molecule used in genetic testing, research, and forensics.
What are the advantages of biopharmaceutical production?
The main advantage is high yield during production.
What are the disadvantages of biopharmaceutical production?
Disadvantages include the potential for endotoxins and differences in post-translational processing compared to mammalian cells.
What are some alternative expression systems for producing biopharmaceuticals?
Alternative expression systems include Chinese hamster ovary cells, tobacco plants, edible plants, and transgenic cattle.
What problems are associated with first-generation monoclonal antibodies?
First-generation monoclonal antibodies can provoke an immune response, have a short half-life, and are less effective at activating human complement.
How can the issues with first-generation monoclonal antibodies be addressed?
The issues can be addressed by humanizing the antibodies to make them more compatible with the human immune system.
What is the purpose of using oligonucleotides in gene therapy?
Oligonucleotides are used as an alternative approach to deliver entire genes, particularly in the form of short antisense oligonucleotides.
What is gene therapy?
Gene therapy is a strategy that involves delivering a functioning copy of a defective gene to a target cell using a suitable vector.
What is the purpose of Gendicine?
Gendicine is used to treat head and neck cancer by replacing a faulty p53 protein.
What is Glybera used for?
Glybera is an adeno-associated virus construct that delivers a correct copy of the lipoprotein lipase gene to patients suffering from lipoprotein lipase deficiency.
What is Strimvelis used for?
Strimvelis is an ex-vivo gene therapy used to treat severe combined immunodeficiency (SCID) due to adenosine deaminase deficiency.
What is the main advantage of using biopharmaceuticals over traditional small molecule drugs?
Biopharmaceuticals are more complex and are derived from biological sources, offering targeted and often more effective treatments, particularly for genetic diseases.
What is one of the most useful applications of oligonucleotides in medicine?
One of the most useful applications of oligonucleotides is in gene therapy, particularly using antisense oligonucleotides to correct genetic defects.
Why are plants like tobacco used in biopharmaceutical production?
Plants, such as tobacco, can be used in biopharmaceutical production because they offer a low-cost, scalable, and biologically compatible method for producing complex biological molecules.
What is a key challenge in the production of biopharmaceuticals using alternative expression systems?
A key challenge is the potential for endotoxins in the final product and differences in post-translational processing when compared to mammalian cells, which could affect the drug’s efficacy and safety.
What are the potential risks of endotoxins in biopharmaceutical production?
Endotoxins can trigger immune responses, leading to fever, inflammation, and other complications. They can pose a serious safety risk if not properly removed during the production process.
Why are gene therapy products like Gendicine, Glybera, and Strimvelis considered groundbreaking in medicine?
These gene therapies offer potential cures for rare, previously untreatable genetic disorders by delivering functional copies of defective genes, thus addressing the root cause of the disease rather than just managing symptoms.
What is the role of vectors in gene therapy?
Vectors, typically viruses or other delivery systems, are used in gene therapy to deliver the functioning copy of a defective gene to target cells, enabling the expression of the correct protein or gene function.
