Nucleic Acid Drug Delivery Technologies: Players, products & prospects to 2018 (Volume III)

Publication Date	June 2009
Publisher	Espicom
Product Type	Report
Pages	110
ISBN Number	978-1-85822-353-7
Product Code	ESP00618

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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
VOLUME III
NUCLEIC ACID DELIVERY
VOLUME IV
TARGETED DELIVERY

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 pr?(C)cis 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.
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-Synth?(C)labo, 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.

Content

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
  - Nucleic acid technology motivators
- Motivators
  - Nucleic acid technology resistors
  - Resistors
Advanced Nucleic Acid Delivery Technologies
- Types of nucleic acid-based products under development by the pharma industry
  - Viral vector delivery of nucleic acids
    - Schematic of gene therapy utilising an AAV vector
    - Schematic of gene therapy utilising HVJ envelope - virosome
  - Bacterial vector delivery of nucleic acids
  - Non-viral vector delivery of nucleic acids
  - Chemical Technologies
    - Stable nucleic acid-lipid particles
    - Targeted nanoparticles
  - Physical Technologies
    - Schematic of Zosano Pharma's Patch Technology
    - Electroporation technology - PassPort Delivery System
    - Advantages and disadvantages of nucleic acid platforms
- Current therapeutic applications for the viral and non-viral delivery of nucleic acids
- Leading nucleic acid based products in development
  - Nucleic acid-based approved/filed drugs
  - Selection of gene therapies in clinical development
  - Selection of RNA therapeutics in clinical development
  - Selection of DNA vaccines in clinical development
- Future commercial applications & opportunities
Strategies of Drug Delivery Companies
- Leading nucleic acid drug delivery companies
  - Leading nucleic acid drug delivery specialists
- Partnerships & acquisitions
  - Strategic developmental & commercialisation partnerships
  - Mergers, acquisitions & joint ventures
    - Recent partnerships & acquisitions in the nucleic acid field
- Intellectual Property
  - Nucleic acid patents
The Viral and Non-Viral Vector Delivery Market
- Now 2009
  - Approved nucleic acid product sales
- Future 2018
  - Novel nucleic acid product sales
  - Global controlled release product sales
Competitor Analysis
- Movers & Shakers: Cutting edge or losing their grip
  - Movers & shakers in nucleic acid drug delivery systems
- COMPANY PROFILES
  - Drug Delivery Technologies: Nucleic Acid
- Alnylam Pharmaceuticals (www alnylam com )
  - 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
    - Alnylam's product pipeline
  - Sales forecasts of products utilising the delivery technology
    - Alnylam's sales forecasts
  - Competitor Ratio Analysis
  - AlphaVax, Inc. (www alphavax com)
  - Proprietary delivery technology
    - The alphavaccine platform system
  - Applications in the pharma industry
  - Management/technical expertise
  - Competition within the delivery technology sector
  - Products in development/approved utilising the delivery technology
    - AlphaVax' product pipeline
  - Sales forecasts of products utilising the delivery technology
    - AlphaVax' sales forecasts
  - Competitor Ratio Analysis
  - Altea Therapeutics, Inc (www alteatherapeutics com)
  - Proprietary delivery technology
    - Altea Therapeutics' Passport Patch
  - Applications in the pharma industry
  - Management/technical expertise
  - Competition within the delivery technology sector
  - Products in development/approved utilising the delivery technology
    - Altea Therapeutics' product pipeline
  - Sales forecasts of products utilising the delivery technology
  - Competitor Ratio Analysis
  - AP Pharma, Inc (www appharma com)
  - 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
    - AP Pharma's product pipeline
  - Sales forecasts of products utilising the delivery technology
  - Competitor Ratio Analysis
  - ArmaGen Technologies (www armagen com)
  - Proprietary delivery technology
    - ArmaGen's BBB delivery technology
  - Applications in the pharma industry
  - Management/technical expertise
  - Competition within the delivery technology sector
  - Products in development/approved utilising the delivery technology
    - ArmaGen Technologies' product pipeline
  - Sales forecasts of products utilising the delivery technology
  - Competitor Ratio Analysis
  - BioDelivery Sciences International (www biodeliverysciences com)
    - Drug Delivery Technologies: Nucleic Acid
  - Proprietary delivery technology
    - Bioral Technology
    - Direct membrane fusion
  - Applications in the pharma industry
  - Management/technical expertise
  - Competition within the delivery technology sector
  - Products in development/approved utilising the delivery technology
    - BDSI's product pipeline
  - Sales forecasts of products utilising the delivery technology
  - Competitor Ratio Analysis
  - Calando Pharmaceuticals (www calandopharma com)
  - Proprietary delivery technology
    - Schematic of cyclodextrin polymeric nanoparticles
  - Applications in the pharma industry
  - Management/technical expertise
  - Competition within the delivery technology sector
  - Products in development/approved utilising the delivery technology
    - Calando's product pipeline
  - Sales forecasts of products utilising the delivery technology
    - Calando's sales forecasts
  - Competitor Ratio Analysis
  - Cequent Pharmaceuticals (www cequentpharma com)
  - Proprietary delivery technology
  - Application in the pharma industry
  - Management/technical expertise
  - Competition within the delivery technology sector
  - Products in development/approved utilising the delivery technology
    - Cequent's product pipeline
  - Sales forecasts of products utilising the delivery technology
    - Cequent's sales forecasts
  - Competitor Ratio Analysis
  - Copernicus Therapeutics (www cgsys com)
  - Proprietary delivery technology
    - Uptake and trafficking of condensed nanoparticle DNA
  - Applications in the pharma industry
  - Management/technical expertise
  - Competition within the delivery technology sector
  - Products in development/approved utilising the delivery technology
    - Copernicus' product pipeline
  - Sales forecasts of products utilising the delivery technology
  - Competitor Ratio Analysis
  - Diatos SA (www diatos com)
  - 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
    - Diatos' product pipeline
  - Sales forecasts of products utilising the delivery technology
  - Competitor Ratio Analysis
  - DOR BioPharma, Inc (www dorbiopharma com)
    - Drug Delivery Technologies: Nucleic Acid
  - 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
    - DOR's product pipeline
  - Sales forecasts of products utilising the delivery technology
  - Competitor Ratio Analysis
  - Expression Genetics, Inc (www egencorp com)
  - Proprietary delivery technology
    - TheraPlas Technology Platform
    - TheraSilence Technology Platform
  - Applications in the pharma industry
  - Management/technical expertise
  - Competition within the delivery technology sector
  - Products in development/approved utilising the delivery technology
    - EGEN's product pipeline
  - Sales forecasts of products utilising the delivery technology
    - EGEN's sales forecasts
  - Competitor Ratio Analysis
  - Inovio Biomedical Corporation (www inovio com)
  - Proprietary delivery technology
    - Inovio's gene delivery platform
    - Inovio's MedPulser DNA Delivery System
    - VGX' Cellectra Technology
  - Applications in the pharma industry
  - Management/technical expertise
  - Competition within the delivery technology sector
  - Products in development/approved utilising the delivery technology
    - Inovio's product pipeline
  - Sales forecasts of products utilising the delivery technology
    - Inovio's sales forecasts
  - Competitor Ratio Analysis
  - MaxCyte, Inc (www maxcyte com)
  - Proprietary delivery technology
    - MaxCyte's GT exvivo cell loading system
  - Applications in the pharma industry
  - Management/technical expertise
  - Competition within the delivery technology sector
  - Products in development/approved utilising the delivery technology
    - MaxCyte's product pipeline
  - Sales forecasts of products utilising the delivery technology
  - Competitor Ratio Analysis
  - Nanotherapeutics (www nanotherapeutics com)
  - 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
    - Nanotherapeutics' product pipeline
  - Sales forecasts of products utilising the delivery technology
    - Drug Delivery Technologies: Nucleic Acid
  - Competitor Ratio Analysis
  - NeoPharm (www neopharm com)
  - Proprietary delivery technology
  - Management/technical expertise
  - Competition within the delivery technology sector
  - Products in development/approved utilising the delivery technology
    - NeoPharm's product pipeline
  - Sales forecasts of products utilising the delivery technology
  - Competitor Ratio Analysis
  - Novagali Pharma SA (www novagali com)
  - Proprietary delivery technology
  - Management/technical expertise
  - Competition within the delivery technology sector
  - Products in development/approved utilising the delivery technology
    - Novagali's product pipeline
  - Sales forecasts of products utilising the delivery technology
  - Competitor Ratio Analysis
  - Oxford Biomedica (www oxfordbiomedica com)
  - Proprietary delivery technology
    - Oxford Biomedica's LentiVector system
  - Management/technical expertise
  - Competition within the delivery technology sector
  - Products in development/approved utilising the delivery technology
    - Oxford Biomedica's product pipeline
  - Sales forecasts of products utilising the delivery technology
    - Oxford Biomedica's sales forecasts
  - Competitor Ratio Analysis
  - PCI Biotech (www pcibiotech com)
  - Proprietary delivery technology
    - PCI enhances siRNA's gene silencing effect and improves gene delivery
  - Applications in the pharma industry
    - Potential applications for improving the delivery of a range of drug types
  - Management/technical expertise
  - Competition within the delivery technology sector
  - Products in development/approved utilising the delivery technology
    - PCI's product pipeline
  - Sales forecasts of products utilising the delivery technology
  - Competitor Ratio Analysis
  - Silence Therapeutics plc (www silence-therapeutics com)
  - 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
    - Silence Therapeutics' product pipeline
  - Sales forecasts of products utilising the delivery technology
    - Silence Therapeutics' sales forecasts
  - Competitor Ratio Analysis
  - Targeted Genetics Corporation (www targetedgenetics com)
  - Proprietary delivery technology
  - Applications in the pharma industry
    - Drug Delivery Technologies: Nucleic Acid
  - Management/technical expertise
  - Competition within the delivery technology sector
  - Products in development/approved utilising the delivery technology
    - TGEN's product pipeline
  - Sales forecasts of products utilising the delivery technology
    - Targeted Genetics sales forecasts
  - Competitor Ratio Analysis
  - Tekmira Pharmaceuticals Corporation (www tekmirapharm com)
  - 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
    - Tekmira's product pipeline
  - Sales forecasts of products utilising the delivery technology
    - Tekmira's sales forecasts
  - Competitor Ratio Analysis
  - TissueGene, Inc (www tissuegene com)
  - Proprietary delivery technology
    - Manufacturing TissueGene-C cell mediated gene therapies
  - Applications in the pharma industry
    - Application of TissueGene-C cell mediated gene therapy in orthopaedics
  - Management/technical expertise
  - Competition within the delivery technology sector
  - Products in development/approved utilising the delivery technology
    - TissueGene's product pipeline
  - Sales forecasts of products utilising the delivery technology
  - Competitor Ratio Analysis
  - Vical, Inc. (www vical com)
  - Proprietary delivery technology
    - Schematic of p-DNA delivery and utilisation in DNA vaccines
  - Applications in the pharma industry
  - Management/technical expertise
  - Competition within the delivery technology sector
  - Products in development/approved utilising the delivery technology
    - Vical's product pipeline
  - Sales forecasts of products utilising the delivery technology
    - Vical's sales forecasts
  - Competitor Ratio Analysis
  - Zosano Pharma, Inc (www zosanopharma com )
  - Proprietary delivery technology
    - ZP Patch Technology
  - Applications in the pharma industry
  - Management/technical expertise
  - Competition within the delivery technology sector
  - Products in development/approved utilising the delivery technology
    - Zosano Pharma's product pipeline
  - Sales forecasts of products utilising the delivery technology
  - Competitor Ratio Analysis
Appendix
- Global nucleic acid approved drugs sales 2008-2018
- Drug Delivery Technologies: Nucleic Acid
Appendix
- Global nucleic acid developmental drugs sales 2008-2018
Appendix
- Competitor Ratio Analysis Criteria
  - Key Nucleic acid delivery companies by 2018
Glossary
Acronyms
Endnotes