Lecture notes
Biologicals and Gene Therapy
- Institution
- University Of Reading (UoR)
Biological Products, Routes of Delivery, Intracellular Targets, Therapeutic Proteins, Growth Factors, Enzymes, Gene Therapy, Historical Timeline,
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Pharmacology and ToxicologyWeek 5
Dr. Mark Dallas
Biologicals & Gene Therapy
- Biological products; vaccines/ blood components/ allergenics / somatic cells/ gene
therapy / recombinant therapeutic proteins
- Biologicals composed of sugars/ proteins / nucleic acids/ complex combos of cells &
tissues
o Isolated from natural sources [human/ animal/ microorganism]
o Produced by biotech [gene based/ cellular biologics= biomedical research/
only cure]
- Biopharmaceuticals include proteins/ antibodies/ oligonucleotides use as drugs
o First generation; copies of endogenous proteins / antibodies produced by
recombinant DNA
o Second generation; “engineered” to improve performance of protein/ antibody.
- Recombinant DNA tech;
1. Gene of interest identified and insert foreign fragment into plasmid vector
[contains cDNA version of mRNA of required protein]
2. Transfect into host cell, replicate and harvest
- Design and use of Biologicals
o Potency/ Specificity/ minimal side effect
o Delivery; cant be delivered oral/ cant cross blood-brain membrane [required
surgery] / broken down by ubiquitous proteases [rapidly metabolised if
injected]
o Manufacture; cost more/ low yield/ purification/ quality control
o Stability; proteins unstable [sensitive to heat/light] / storage and shelf life
usually lower
o Investment in formulation and production.
- Routes of Delivery;
1. Buccal delivery [Film/spray]; Insulin/ Interferon [Clinical trials]
2. Ocular Delivery [eye drops/ injection] VEGF-targeted Fed/ igGmAB [Approved]
3. Nasal Delivery [sprays]; salmon calcitonin [Approved]
4. Oral Delivery [pill/capsule]; calcitonin/ insulin/ exenatide/ octreotide [Clinical]
5. Sublingual Delivery [tablet]; desmopressin [approved]
6. Transdermal Delivery [patch/cream/injection]; PTH/insulin [Clinical Trial]
7. Pulmonary Delivery [aerosol/ dry powder]; Insulin/ Dornase Afa [Approved]
insulin under clinical trial
8. Vaginal Delivery [Creams/gels]; LHRH analogue [Clinical trial]
9. Rectal Delivery [suppositories]; n/a
- Intracellular targets;
1. Mitochondrial/ subcellular targets;
o Lysosomal enzymes/ heat shock proteins [HSP90]/ mitochondrial carriers/
apoptotic factors/ transporters/ intracellular receptors
2. Cytoplasmic targets;
o Kinases/ other enzymes
3. Nuclear Targets;
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