Nucleic Acid DeliveryThe nucleic acid delivery market is maturing with over 1,450 clinical trials underway worldwide. The majority of these are in early clinical development (approximately 60%) with just over 3% in Phase III trials. Our analysis indicates that up to 35 novel nucleic acid products could reach the market within our forecasting period (2008-2018). However the harsh economic environment may force companies to place promising drugs on hold as they focus on lead projects rather than multiple programmes. To date, only a handful of nucleic acid-based products have been approved for human prescription and these products rely on delivery technologies in one shape or form. We estimate the nucleic acid market was worth just over US$80 million globally in 2008.By 2018 we anticipate the nucleic acid technology market will have matured as many of the late-stage clinical programmes come to fruition and drug delivery companies continue to overcome issues surrounding its safety and efficiency. The development of nucleic acid delivery systems will help to improve specificity whilst the development of novel polymers and lipopolymer complexes will help to carry larger DNA payloads in order to target cells and make multigene targeting a reality. We forecast that nucleic acid products in development from 11 key companies could generate additional sales of nearly US$2.3 billion by 2018. These products utilise a range of delivery platforms including viral to non-viral delivery systems, and cover gene therapies, gene agents and DNA vaccines for a variety of targets including cancer, cardiovascular and infectious diseases.Catalysts for changeImprovements in our understanding into the underlying genetic causes of disease as well as the uptake of new mechanisms of modulating DNA such as RNA interference which will help to fuel market growth.The development of a new generation of DNA vaccines that will provoke strong immunogenic responses and will help to determine whether this has been a sound investment for the pharma industry.Launch of the first siRNA-based therapeutics (although progress has been dampened following the recent termination of Opko Health's leading eye treatment at the Phase III development stage). We anticipate companies will pursue other conditions such as infectious diseases and cancer where robust preclinical studies have already been performed and where drug delivery platforms have been adopted rather than the use of naked siRNAs.This report is part of a major new 4-volume strategic analysisDrug Delivery Technologies:Players, Products & Prospects to 2018Focusing on the sectors that are driving growthVOLUME ICONTROLLED- RELEASEVOLUME IINANOTECHNOLOGYVOLUME IIINUCLEIC ACID DELIVERYVOLUME IVTARGETED DELIVERYClick here for details of the full report >> CONTENTSEXECUTIVE SUMMARY & INTRODUCTION - Overview of delivery technologies for genetic material: past, present & future - Leading technology platforms for the delivery of genetic material - Nucleic acid delivery: motivators & resistorsADVANCED NUCLEIC ACID DELIVERY TECHNOLOGIES - Types of nucleic acid-based products under development by the pharma industry - Viral vector delivery of nucleic acids - Bacterial vector delivery of nucleic acids - Non-viral vector delivery of nucleic acids - Chemical Technologies - Physical Technologies - Current therapeutic applications for the viral and non-viral delivery of nucleic acids - Leading nucleic acid-based products in development - Future commercial applications & opportunitiesSTRATEGIES OF DRUG DELIVERY COMPANIES - Leading nucleic acid drug delivery companies - Strategic developmental & commercialisation partnerships - Mergers, acquisitions & joint ventures - Intellectual PropertyTHE VIRAL AND NON-VIRAL VECTOR DELIVERY MARKET - Now 2009: Approved nucleic acid product sales - Future 2018: Novel nucleic acid product sales and Global nucleic acid product sales COMPETITOR ANALYSISDetailed analysis of leading companies cove

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Nucleic Acid Drug Delivery Technologies: Players, products &
prospects to 2018 (Volume III)
Published on June 2009
Report Summary
Nucleic Acid Delivery
The nucleic acid delivery market is maturing with over 1,450 clinical trials underway worldwide. The majority of these are in early
clinical development (approximately 60%) with just over 3% in Phase III trials. Our analysis indicates that up to 35 novel nucleic acid
products could reach the market within our forecasting period (2008-2018). However the harsh economic environment may force
companies to place promising drugs on hold as they focus on lead projects rather than multiple programmes. To date, only a handful
of nucleic acid-based products have been approved for human prescription and these products rely on delivery technologies in one
shape or form. We estimate the nucleic acid market was worth just over US$80 million globally in 2008.
By 2018 we anticipate the nucleic acid technology market will have matured as many of the late-stage clinical programmes come to
fruition and drug delivery companies continue to overcome issues surrounding its safety and efficiency. The development of nucleic
acid delivery systems will help to improve specificity whilst the development of novel polymers and lipopolymer complexes will help to
carry larger DNA payloads in order to target cells and make multigene targeting a reality. We forecast that nucleic acid products in
development from 11 key companies could generate additional sales of nearly US$2.3 billion by 2018. These products utilise a range
of delivery platforms including viral to non-viral delivery systems, and cover gene therapies, gene agents and DNA vaccines for a
variety of targets including cancer, cardiovascular and infectious diseases.
Catalysts for change
Improvements in our understanding into the underlying genetic causes of disease as well as the uptake of new mechanisms of
modulating DNA such as RNA interference which will help to fuel market growth.
The development of a new generation of DNA vaccines that will provoke strong immunogenic responses and will help to determine
whether this has been a sound investment for the pharma industry.
Launch of the first siRNA-based therapeutics (although progress has been dampened following the recent termination of Opko
Health's leading eye treatment at the Phase III development stage). We anticipate companies will pursue other conditions such as
infectious diseases and cancer where robust preclinical studies have already been performed and where drug delivery platforms have
been adopted rather than the use of naked siRNAs.
This report is part of a major new 4-volume strategic analysis
Drug Delivery Technologies:
Players, Products & Prospects to 2018
Focusing on the sectors that are driving growth
VOLUME I
CONTROLLED- RELEASE
VOLUME II
NANOTECHNOLOGY
Nucleic Acid Drug Delivery Technologies: Players, products & prospects to 2018 (Volume III) Page 1/7

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VOLUME III
NUCLEIC ACID DELIVERY
VOLUME IV
TARGETED DELIVERY
Click here for details of the full report >>
CONTENTS
EXECUTIVE SUMMARY & INTRODUCTION
- Overview of delivery technologies for genetic material: past, present & future
- Leading technology platforms for the delivery of genetic material
- Nucleic acid delivery: motivators & resistors
ADVANCED NUCLEIC ACID DELIVERY TECHNOLOGIES
- Types of nucleic acid-based products under development by the pharma industry
- Viral vector delivery of nucleic acids
- Bacterial vector delivery of nucleic acids
- Non-viral vector delivery of nucleic acids
- Chemical Technologies
- Physical Technologies
- Current therapeutic applications for the viral and non-viral delivery of nucleic acids
- Leading nucleic acid-based products in development
- Future commercial applications & opportunities
STRATEGIES OF DRUG DELIVERY COMPANIES
- Leading nucleic acid drug delivery companies
- Strategic developmental & commercialisation partnerships
- Mergers, acquisitions & joint ventures
- Intellectual Property
THE VIRAL AND NON-VIRAL VECTOR DELIVERY MARKET
- Now 2009: Approved nucleic acid product sales
- Future 2018: Novel nucleic acid product sales and Global nucleic acid product sales
COMPETITOR ANALYSIS
Detailed analysis of leading companies covering:
- Proprietary delivery technology
- Applications in the pharma industry
- Management/technical expertise
- Competition within the delivery technology sector
- Products in development/approved utilising the delivery technology
- Sales forecasts of products utilising the delivery technology
- Competitor ratio analysis
Companies analysed
- Alnylam Pharmaceuticals, Inc.
- AlphaVax, Inc.
- Altea Therapeutics, Inc.
- AP Pharma, Inc.
- ArmaGen Technologies, Inc.
- BioDelivery Sciences International, Inc.
- Calando Pharmaceuticals, Inc.
- Cequent Pharmaceuticals, Inc.
- Copernicus Therapeutics, Inc.
Nucleic Acid Drug Delivery Technologies: Players, products & prospects to 2018 (Volume III) Page 2/7

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- Diatos SA, Inc.
- DOR BioPharma, Inc
- Expression Genetics, Inc
- Inovio Biomedical Corporation
- MaxCyte, Inc
- Nanotherapeutics, Inc.
- NeoPharm, Inc.
- Novagali Pharma, S.A.
- Oxford Biomedica, Plc.
- PCI Biotech ASA.
- Silence Therapeutics, Plc.
- Targeted Genetics Corporation
- TissueGene, Inc.
- Vical, Inc.
- Zosano Pharma, Inc.
APPENDICIES
- Global nucleic acid approved drug sales 2008-2018
- Global nucleic acid development drug sales 2008-2018
- Key nucleic acid delivery companies by 2018
Unique company & technology evaluation
It is vital that management information is reliable, current and insightful. Today, product and company assessment must be seen in a
wider competitive and market environment.
In this report we present a précis on each of the leading delivery specialists and potential new delivery platforms and have identified
their key attributes, based on a number of criteria including:
Proprietary delivery technology
Applications of the delivery technology within the pharmaceutical industry
Management/technical expertise of the delivery specialist
Competition within the delivery technology sector
Products in development/approved utilising the delivery technology
We have established a competitor ratio analysis based on each of the criteria mentioned above. Each criterion attracts an award of up
to 10 points, with a maximum score of 50, thus enabling the establishment of an Espicom 'ranking table' in each segment of the
market, dependent on their relative attributes.
Multi-point evaluation and scoring assessment
Pharma drugs that utilise drug delivery systems have been evaluated based on a number of parameters, including:
The technology
Comparison of delivery platforms within each area including: activated, degradation or diffusion for controlled-release systems;
nano-engineered (nanocrystals, nanoparticle, nanosuspension) or nanocarriers (carbon/ceramic, dendrimer, liposome, micelle,
polymeric nanoparticles) for nano-enabled delivery; viral or non-viral platforms for nucleic acid delivery and active or passive platforms
for targeted delivery systems
The application of the technology within a number of therapy areas
Route of delivery (oral, parenteral, transdermal, implantable), delivery of small molecules/macromolecules and vaccines, additional
features such as bioerodible materials etc
The current status of the technology in clinical development or following approval
Number of approved and marketed products, clinical or preclinical development candidates, single or multiple products.
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Competition within the technology arena
Alternative drug delivery systems to technologies under evaluation, advantages and disadvantages over competitors.
The drug delivery specialist's expertise
Number of drug delivery systems in development, strategic alliances, financial position, marketing presence.
The product portfolio
Drug delivery systems under evaluation, products in development, commercialisation/development partnerships.
Potential drug sales
Based on key events such as market, competition, patent expiry, time of entry into the market.
About the Author
Dr Cheryl Barton is a highly regarded independent consultant with over 16 years research and business analysis experience.
Following her senior research positions in academia and seven years with Merck, in which she was responsible for research projects
ranging from Alzheimer's disease to schizophrenia. Dr Barton subsequently joined Dutch investment bank ABN Amro NV as a senior
equity analyst where she was lead analyst on major pharmaceutical companies such as Roche and Sanofi-Synthélabo, and assessed
the potential impact of new drug development on European Stocks.
In 2002, Dr Barton founded a consulting business (www.Pharmavision.co.uk) to provide independent, tailor-made, pharmaceutical
thematic research to investment houses and pharmaceutical companies. The research reports combine independent scientific
analysis with patient-based models to forecast the potential sales growth of key drugs in clinical development.
Table of Content
CONTENTS
EXECUTIVE SUMMARY & INTRODUCTION
- Overview of delivery technologies for genetic material: past, present & future
- Leading technology platforms for the delivery of genetic material
- Nucleic acid delivery: motivators & resistors
ADVANCED NUCLEIC ACID DELIVERY TECHNOLOGIES
- Types of nucleic acid-based products under development by the pharma industry
- Viral vector delivery of nucleic acids
- Bacterial vector delivery of nucleic acids
- Non-viral vector delivery of nucleic acids
- Chemical Technologies
- Physical Technologies
- Current therapeutic applications for the viral and non-viral delivery of nucleic acids
- Leading nucleic acid-based products in development
- Future commercial applications & opportunities
STRATEGIES OF DRUG DELIVERY COMPANIES
- Leading nucleic acid drug delivery companies
- Strategic developmental & commercialisation partnerships
- Mergers, acquisitions & joint ventures
- Intellectual Property
THE VIRAL AND NON-VIRAL VECTOR DELIVERY MARKET
- Now 2009: Approved nucleic acid product sales
- Future 2018: Novel nucleic acid product sales and Global nucleic acid product sales
Nucleic Acid Drug Delivery Technologies: Players, products & prospects to 2018 (Volume III) Page 4/7

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COMPETITOR ANALYSIS
Detailed analysis of leading companies covering:
- Proprietary delivery technology
- Applications in the pharma industry
- Management/technical expertise
- Competition within the delivery technology sector
- Products in development/approved utilising the delivery technology
- Sales forecasts of products utilising the delivery technology
- Competitor ratio analysis
Companies analysed
- Alnylam Pharmaceuticals, Inc.
- AlphaVax, Inc.
- Altea Therapeutics, Inc.
- AP Pharma, Inc.
- ArmaGen Technologies, Inc.
- BioDelivery Sciences International, Inc.
- Calando Pharmaceuticals, Inc.
- Cequent Pharmaceuticals, Inc.
- Copernicus Therapeutics, Inc.
- Diatos SA, Inc.
- DOR BioPharma, Inc
- Expression Genetics, Inc
- Inovio Biomedical Corporation
- MaxCyte, Inc
- Nanotherapeutics, Inc.
- NeoPharm, Inc.
- Novagali Pharma, S.A.
- Oxford Biomedica, Plc.
- PCI Biotech ASA.
- Silence Therapeutics, Plc.
- Targeted Genetics Corporation
- TissueGene, Inc.
- Vical, Inc.
- Zosano Pharma, Inc.
APPENDICIES
- Global nucleic acid approved drug sales 2008-2018
- Global nucleic acid development drug sales 2008-2018
- Key nucleic acid delivery companies by 2018
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