Emerging Drug Discovery Technologies

Transcription

1 HEALTHCARE Emerging Drug Discovery Technologies Building competitive advantage through lab-on-a-chip, nanotechnology and RNAi By Dr. Nelesh Patel Table of Contents

2 Dr. Nelesh Patel, B.Sc., Ph.D. Dr. Nelesh Patel, a former Post-Doctoral Research Associate and Healthcare Analyst has acquired more than 5 years experience in scientific research and has built relationships/contacts with major medical device/biotechnology and pharmaceutical companies. Dr. Patel is currently set-up as an independent consultant specializing in the production of custom and syndicated market intelligence deliverables to a range of medical device, pharmaceutical and biotechnology markets. His current research projects include orthopedic medical devices, the role of nanotechnology in medicine and emerging trends in drug discovery. Dr. Patel has published more than 10 scientific peer reviewed journal papers and has delivered award-winning presentations at medical device/biotechnology conferences. He graduated with a BSc (Hons) in Biomedical Materials Science from the University of Manchester and a PhD in Orthopedic Medical Devices from the University of Cambridge. Copyright 2005 Business Insights Ltd This Management Report is published by Business Insights Ltd. All rights reserved. Reproduction or redistribution of this Management Report in any form for any purpose is expressly prohibited without the prior consent of Business Insights Ltd. The views expressed in this Management Report are those of the publisher, not of Business Insights. Business Insights Ltd accepts no liability for the accuracy or completeness of the information, advice or comment contained in this Management Report nor for any actions taken in reliance thereon. While information, advice or comment is believed to be correct at the time of publication, no responsibility can be accepted by Business Insights Ltd for its completeness or accuracy. ii

3 Table of Contents Emerging Drug Discovery Technologies Executive Summary 10 Introduction 10 Lab-on-a-chip/microfluidics 11 Nanotechnology 12 RNA interference 13 Chapter 1 Introduction 16 Summary 16 Overview 17 A historical overview of the drug discovery process 18 Drug discovery in the 1950s and 1960s 18 Drug discovery in the 1980s 19 Drug discovery today 20 Emerging drug discovery technologies 22 Lab-on-a-chip/microfluidic technologies 22 Nanotechnology 23 RNA interference 24 Report objectives 25 Chapter 2 Lab-on-a-chip/microfluidics 27 Summary 27 Introduction 28 LOC technologies 31 LOC design issues 32 Microarrays 32 Spotted microarrays 33 In situ fabricated arrays DNA applications Protein applications 36 iii

4 The LabChip 37 Lab-on-a-CD 41 Chip-based electrospray system for mass spectroscopy 43 HPLC-on-a-chip 44 Microfluidic PCR 45 Multiplexed capillary electrophoresis 48 Market drivers and restraints 48 Market drivers 49 HTS 49 Reduction of human error 50 Reduced exposure to hazardous materials 50 Reduction in sample requirement and routine tasks 50 Cost savings Market restraints Reduction in demand 51 Reluctance to replace old systems with new technologies 52 Market analysis 52 Competitive structure 54 Market share 54 Aclara BioSciences 55 Advion BioSciences, Inc. 56 Agilent Technologies 57 BioMicro Systems 58 BioTrove Inc. 59 Caliper Life Sciences 60 Cepheid 62 CombiMatrix Corp. 63 Eksigent Technologies 64 Epigem Limited 65 Fluidigm Corp. 66 Gyros AB 67 Nanogen 68 Nanostream Inc. 70 Protiveris 71 Sequenom 71 Zyomyx 72 Future developments 73 Chapter 3 Nanotechnology 77 Summary 77 Introduction 78 Nanotechnologies 79 Applications 81 Drug discovery and drug delivery 81 Biosensing 82 Other applications 82 iv

5 Public opinion 82 Funding 83 Market 84 Nano-enabled drug discovery tools 84 Atomic force microscopy 85 Nano-mass spectroscopy 86 Dip-pen nanolithography 87 Nanoarrays 88 Nanoparticles for drug discovery 90 Quantum dots and gold nanoparticles 92 Nanoshells 95 Nanobarcode particles 97 Nano-enabled drugs 98 Abraxane 98 RenaZorb 98 Antimicrobial emulsions 99 Antioxidants and fullerenes 99 Industry challenges 101 High demands of drug discovery applications 102 Long-term stability of nanomaterials 103 Technical issues in nano-assembly and molecular manufacturing 104 Barriers to collaborations 104 Lack of test standardization 104 Scalability 105 Pharmaceutical companies reluctant to invest in nanotechnology 105 Funding for nanotechnology 105 Market drivers and restraints 106 Market drivers 108 Technological drivers 108 Increased funding 109 Social and economic factors Market restraints Uncertainty 111 Public awareness 111 Environmental concerns 112 Detection of incurable diseases 112 Large expectations 112 Market analysis 113 Competitive structure 114 Market share 115 3DM Inc. 117 Alnis Biosciences Inc. 118 American Pharmaceuticals Partners Inc. 119 BioCrystal Ltd. 120 BioForce NanoSciences Inc. 120 CrystalPlex Corp. 122 C Sixty Inc. 122 Evident Technologies, Inc. 123 v

6 NanBio Corp. 124 Nanosphere 125 PharmaSeq, Inc 126 Quantum Dot Corp. 128 Future developments 129 Chapter 4 RNA interference (RNAi) 133 Summary 133 Introduction 134 Gene silencing 137 Methods for gene silencing 137 Aptamers 138 Ribozymes 138 Peptide nucleic acid (PNA) and PNA-DNA chimeras 140 Antisense oligonucleotides 140 RNA interference (RNAi) 142 RNAi mechanism 142 RNAi approaches 143 sirna 144 ddrnai 144 MicroRNAs (mirnas) 145 Short hairpin RNAs (shrnas) 146 Delivery methods 146 Delivery using chemical transfections 147 Delivery using electroporation 149 Delivery using expression vectors 149 Delivery using cell-penetrating peptides 151 In vivo delivery of sirnas 151 Industry challenges 152 Intellectual property issues 153 Off-target effects 153 Delivery of sirna in vitro and in vivo 154 Toxicity issues from RNAi 154 Pricing issues 155 Efficacy of RNAi 155 Standardization of technologies 156 Proof of therapeutic potential 156 Market drivers and restraints 157 Market drivers 158 Demand for accelerated drug discovery 158 Resolution of intellectual property issues 158 Robust, efficient and potent technology 158 Functional genomics and proteomics studies 159 Vector-based and sirna price decline 159 Reliability 159 vi

7 Market restraints 160 Expensive RNAi reagents 160 Delivery of sirna 160 Failure of antisense drugs 160 Lack of robust clinical data 161 Selection and design of appropriate sirna 161 Toxicity and off-target effects 161 Market analysis 161 Revenue forecasts 161 Trends by geographic region 163 North America 164 Europe 164 Japan 164 Rest of the World 164 Competitive structure 165 Market share analysis 166 Ambion 168 Alnylam Pharmaceuticals, Inc. 168 Benitec Ltd 169 CytRx Corp. 170 Cenix BioScience 170 Devgen 171 Dharmacon, Inc. 172 Eurogentec (EGT) 173 Imgenex Corp. 173 Immusol Inc. 174 Invitrogen Corp. 174 InvivoGen 174 MWG Biotech 175 Proligo 175 Promega 176 Qiagen. 176 Sirna Therapeutics 177 Future developments 178 Chapter 5 Appendix 181 Research methodology and information sources 181 Primary research methodology 181 Secondary research methodology 182 Market sizing 182 Forecasting 182 Index 184 Abbreviations and acronyms 185 vii

8 List of Figures Figure 1.1: Drug development process during the 1950s and 1960s 19 Figure 1.2: Drug development process during the 1980s 20 Figure 1.3: Drug development process today 21 Figure 2.1: The Gyros Bioaffy lab-on-a-cd 42 Figure 2.2: Advion BioSciences ESI Chip 44 Figure 2.3: HPLC-on-a-chip 45 Figure 2.4: Cepheid s GeneXpert cartridges for PCR analysis 47 Figure 2.5: Market drivers and restraints for LOC/microfluidics devices in drug discovery 49 Figure 2.6: Microfluidics/LOC revenue forecasts, Figure 2.7: Microfluidics/lab-on-a-chip market shares, Figure 3.1: Expression of cells using QDots 94 Figure 3.2: Binding of Nanosphere s nanoparticle probe to a selected genetic or proteomic target 95 Figure 3.3: Nano-enabled drug discovery industry challenges 102 Figure 3.4: Market drivers and restraints for nano-enabled drug discovery 107 Figure 3.5: Nano-enabled drug discovery revenue forecasts, Figure 3.6: Nanoenabled drug discovery market shares, Figure 4.7: RNAi pathways 143 Figure 4.8: Industry challenges faced by RNAi industry participants 152 Figure 4.9: Market drivers and restraints for RNAi technology 157 Figure 4.10: RNAi revenue forecasts and growth rates, Figure 4.11: RNAi revenues by geographic region, Figure 4.12 RNAi market shares, List of Tables Table 2.1: Microfluidics/LOC revenue forecasts, Table 3.2: Nano-enabled drug discovery revenue forecasts, Table 4.3: RNAi revenue forecasts, Table 5.4: Abbreviations and acronyms, A-L 185 Table 5.5: Abbreviations and acronyms, M-Z 186 viii