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Drug discovery and development
Introduction
 In the past most drugs have been discovered either by
identifying the active ingredient from traditional
remedies or by serendipitous discovery.
 But now we know diseases are controlled at molecular
and physiological level.
 Also shape of an molecule at atomic level is well
understood.
 Information of Human Genome
Drug discovery and development
History of Drug Discovery :
Pre 1919
Herbal Drugs
Serendiptious discoveries
1920s, 30s
Vitamins
Vaccines
1940s
Antibiotic Era
R&D Boost due to WW2
1950s
New technology,
Discovery of DNA
1960s
Breakthrough in Etiology
1970s
Rise of Biotechnology
Use of IT
1980s
Commercialization of Drug
Discovery
Combinatorial Chemistry
1990s
Robotics
Automation
Drug Discovery methods
 Drugs Discovery methods:
 Random Screening
 Molecular Manipulation
 Molecular Designing
 Drug Metabolites
 Serendipity
Steps Of Drug Discovery
Target Selection
Lead
Discovery
Medicinal
Chemistry
In Vitro
Studies
In Vivo
Studies
Clinical
Trials
Cellular &
Genetic Targets
Genomics
Proteomics
Bioinformatics
Synthesis and
Isolation
Combinatorial
Chemistry
Assay
Development
High
Throughput
Screening
Library
Development
SAR Studies
In Silico
Screening
Chemical
Synthesis
Drug Affinity
and Selectivity
Cell Disease
Models
MOA
Lead Candidate
Refinement
Animal models
of Disease States
Behavioural
Studies
Functional
Imaging
Ex-Vivo
Studies
Phase-I
Phase-II
Phase-III
Phase-IV
Gene Therapy
 Technique for correcting defective
genes.
 It is the process of inserting genes
into cells to treat diseases.
 Gene therapy is used to correct a
deficient phenotype.
Recent Developments
 Nanotechnology + gene therapy yielded treatment to
torpedo cancer
 Results of world's first gene therapy for inherited
blindness show sight improvement
 New Method of Gene Therapy Alters Immune Cells
for Treatment of Advanced Melanoma
 Dual Gene Therapy Suppresses Lung Cancer in
Preclinical Test
Orphan Drugs:
 An orphan drug is a pharmaceutical agent that has been
developed specifically to treat a rare medical condition,
the condition itself being referred to as an orphan disease.
 National Organization for Rare
Disorders
 European Organization for Rare
Diseases
Advantages:
 Tax incentives.
 Enhanced patent protection and marketing rights.
 Clinical research financial subsidization.
 Rise in research and developmen.
 Crown Corporation.
Orphan Drugs Act:
 4th January 1983
 6000 Orphan Diseases
 Unprofitable Drug Development
 Affecting < 2,00,000 Persons
 Orphan Drug Status to 1,090
Drugs
 1985 Amendment- Marketing
Exclusivity
Tourette
Syndrome
An Orphan
Disease
Drug discovery and development

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Drug discovery and development

  • 2. Introduction In the past most drugs have been discovered either by identifying the active ingredient from traditional remedies or by serendipitous discovery. But now we know diseases are controlled at molecular and physiological level. Also shape of an molecule at atomic level is well understood. Information of Human Genome
  • 4. History of Drug Discovery : Pre 1919 Herbal Drugs Serendiptious discoveries 1920s, 30s Vitamins Vaccines 1940s Antibiotic Era R&D Boost due to WW2 1950s New technology, Discovery of DNA 1960s Breakthrough in Etiology 1970s Rise of Biotechnology Use of IT 1980s Commercialization of Drug Discovery Combinatorial Chemistry 1990s Robotics Automation
  • 5. Drug Discovery methods Drugs Discovery methods: Random Screening Molecular Manipulation Molecular Designing Drug Metabolites Serendipity
  • 6. Steps Of Drug Discovery Target Selection Lead Discovery Medicinal Chemistry In Vitro Studies In Vivo Studies Clinical Trials Cellular & Genetic Targets Genomics Proteomics Bioinformatics Synthesis and Isolation Combinatorial Chemistry Assay Development High Throughput Screening Library Development SAR Studies In Silico Screening Chemical Synthesis Drug Affinity and Selectivity Cell Disease Models MOA Lead Candidate Refinement Animal models of Disease States Behavioural Studies Functional Imaging Ex-Vivo Studies Phase-I Phase-II Phase-III Phase-IV
  • 7. Gene Therapy Technique for correcting defective genes. It is the process of inserting genes into cells to treat diseases. Gene therapy is used to correct a deficient phenotype.
  • 8. Recent Developments Nanotechnology + gene therapy yielded treatment to torpedo cancer Results of world's first gene therapy for inherited blindness show sight improvement New Method of Gene Therapy Alters Immune Cells for Treatment of Advanced Melanoma Dual Gene Therapy Suppresses Lung Cancer in Preclinical Test
  • 9. Orphan Drugs: An orphan drug is a pharmaceutical agent that has been developed specifically to treat a rare medical condition, the condition itself being referred to as an orphan disease. National Organization for Rare Disorders European Organization for Rare Diseases
  • 10. Advantages: Tax incentives. Enhanced patent protection and marketing rights. Clinical research financial subsidization. Rise in research and developmen. Crown Corporation.
  • 11. Orphan Drugs Act: 4th January 1983 6000 Orphan Diseases Unprofitable Drug Development Affecting < 2,00,000 Persons Orphan Drug Status to 1,090 Drugs 1985 Amendment- Marketing Exclusivity Tourette Syndrome An Orphan Disease