Biotechnology-based drugs

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Chapter: Pharmaceutical Drugs and Dosage: Biotechnology-based drugs

Almost all human diseases are the result of inappropriate protein production or due to some structural disorder that impacts protein performance.


Biotechnology-based drugs

Introduction

Almost all human diseases are the result of inappropriate protein production or due to some structural disorder that impacts protein performance. Traditional small molecule drugs are designed to interact with protein molecules that support or cause diseases. Protein drugs seek to replace the defec-tive protein in cases where missing or defective protein is the cause of the disease. Enzyme replacement therapy is a common example of protein drugs that seek to replace inherently defective enzymes. Many severe and debilitat-ing diseases (e.g., diabetes, hemophilia, and cystic fibrosis) and several chronic diseases (i.e., hypertension, ischemic heart disease, asthma, Parkinson’s disease, motor neuron disease, and multiple sclerosis) remain inadequately treated by conventional small molecular weight and protein drugs.

Compared to conventional small molecular weight and protein drugs, nucleic acid medicines are designed to suppress or generate endogenous proteins by acting on or with the transcription and translation mechanisms of formation of proteins from the genetic code. These medicines can be administered to patients by conventional routes, such as direct injection, inhalation, or intravenous injection. Several different approaches are used for turning nucleic acids into therapeutics. Among them, antisense oli-gonucleotides (ODNs), RNA interference (RNAi) technologies, plasmid DNA and virus-based gene therapy approaches are most widely studied. Antisense ODNs and small interfering RNAs (siRNAs) aim at inhibit-ing aberrant protein production, whereas gene therapy aims at using the patient’s somatic cells to produce therapeutic proteins needed for treating genetic or acquired diseases. The promise of these nucleic acid drugs is to allow either the production of therapeutic proteins that may be difficult to administer exogenously or the inhibition of abnormal protein production.

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