Gene Therapy and Biological Drugs

The aim of the subject is to get acquainted the students with tools and methods employed in area of gene therapy and biological drugs focusing on pharmaceutical aspects of the issues. The means of gene therapy and biological drugs generally are presented in terms of their origin, structure, properties, methods of manufacture, and quality control. Students are further informed about current situation in clinical use as well as arising potential of biological and gene therapy. Also limits and risks of these approaches are discussed.


Biological drugs generally

  • definition of biologics
  • differences between classical drugs and biologics
  • classification of biologics
    • peptides and proteins (monoclonal antibodies, enzymes, vaccines)
    • polysaccharides, mucopolysaccharides
    • nucleic acids based biologics
    • combinations, technological adjustments (e.g. macromolecular prodrugs)
  • nomenclature of biologics

Methods of manufacture of biologics and structural predispositions for biological activity

  • biotechnological manufacture of protein-based biologics
  • modification of protein-based drugs structure to increase their biological effect and/or stability
  • biotechnological manufacture of polysaccharide-based drugs
  • principles of chemical synthesis of oligopeptides a oligonucleotides on solid phase
  • oligonucleotide therapeutics – 1st, 2nd and 3rd generations of modifications to improve stability in biological environment and pharmacokinetic properties
  • pharmaceutical-technological and pharmacokinetic properties resulting of structural features of biologics
  • pharmaceutical-technological methods to increase biological activity of biologics
  • modifications of biopolymers for diagnostic reasons

Methods of control and analysis in area of biologic drugs

  • approaches to analytical evaluation of biological drugs
  • most frequently employed methods for analysis of biological drugs
  • evaluation of identity, quality and content of biological drugs

Gene therapy introduction

  • division of gene therapy
  • GMO legislation

Oligonucleotide Mediated Gene Therapy (OMGT)

  • principles of antisense and antigene therapy
  • possibilities, problems and limits of of OMGT
  • antisense drugs in clinical use and trials
  • overview of non-coding RNAs
  • microRNA a siRNA
  • possibilities of therapeutic use of non-coding RNA based modulation of gene expression

Vectors Mediated Gene Therapy (VMGT)

  • possibilities of VMGT
  • types of vectors employed for VMGT
  • ways of preparation of vectors
  • advantages and disadvantages of various types of vectors
  • problems and risks of VMGT
  • DNA vaccines

Aptamers as specific group of therapeutics

  • structural types of aptamers
  • mechanisms of action
  • preparation of oligonucleotide aptamers
  • structural predispositions for biological activity of oligonucleotide aptamers
  • aptamers in clinical use and trials

Therapy realized by monoclonal antibodies (MAB)

  • mechanisms of action
  • constitutional types of antibodies (naked, conjugated, radioactively labelled)
  • chemical and physical-chemical modifications of antibodies to improve their pharmacokinetic properties
  • overview of current therapeutic use ofMAB and other biologics focused on oncology, therapy of rheumatoid arthritis and autoimmune diseases

Targeted therapy of cancer diseases and therapeutic influence on cell signalization processes

  • modern molecular approaches based on biologics and low-molecular inhibitors of cell signalization

Stem cells, cell therapy.

  • types of stem cells (SCs)
  • methods of SCs generation
  • applications of SCs


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