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世界の遺伝子治療市場(2018年まで):治験の失敗、厳格な規制・監視、高いコンプライアンス基準による製品開発の減速

Gene Therapy Market to 2018 - Product Development Slowed by Clinical Failures, Close Regulatory Surveillance and High Compliance Standards

発行 GBI Research 商品コード 245745
出版日 ページ情報 英文 76 Pages
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世界の遺伝子治療市場(2018年まで):治験の失敗、厳格な規制・監視、高いコンプライアンス基準による製品開発の減速 Gene Therapy Market to 2018 - Product Development Slowed by Clinical Failures, Close Regulatory Surveillance and High Compliance Standards
出版日: 2012年06月25日 ページ情報: 英文 76 Pages
概要

当レポートでは、世界各国での遺伝子治療の最新の技術・市場動向について分析し、遺伝子治療の概要や現在までの沿革、市場促進・抑制要因、現在の治験の推進状況、規制環境、関連企業のプロファイルといった情報を盛り込んで、概略以下の構成でお届けします。

第1章 目次

第2章 イントロダクション

第3章 技術概要

  • イントロダクション
    • 遺伝子
    • ゲノム
    • DNA
  • 遺伝子疾患
  • 遺伝子治療
    • 遺伝子治療の種類
  • 遺伝子治療のニーズ
  • 遺伝子治療に使われるベクター
    • ウイルスベクター
    • 非ウイルスベクター
  • 医薬品産業における遺伝子治療の役割
    • 上流工程としての遺伝子治療の活動が、既存の生物学的療法よりも効果的である

第4章 臨床開発の傾向

  • 年度別件数
  • 利用するベクター別の件数
  • 地域別件数
  • 治療分野別件数

第5章 市場環境の特徴

  • 市場促進要因
    • 癌や他の適応症に対する従来型療法の失敗が、高いアンメットニーズを生み出した
    • 先進国での遺伝子治療の導入と、市場成長との釣り合い
    • 致命的な慢性疾患の発症増加と流行
    • 単因子条件での治療の可能性が、以前は未開発だった市場へのアクセスを可能にする
  • 市場抑制要因
    • 厳格な法規制による開発の遅延
    • 安全上の懸念
    • 現在の技術の限界
  • 市場分析
    • 強み
    • 弱み
    • 機会
    • 脅威
  • 現在の市販製品
    • Gendicine
    • Oncorine
    • Rexin-G

第6章 パイプライン分析

  • 研究開発活動の分析
    • フェーズ別
    • 治療領域別
  • パイプライン上の有望な分子
    • Collategene
    • Allovectin-7
    • Generx
  • 最終段階で治験に失敗した製品
    • Glybera
    • Cerepro
    • NV1FGF
    • TNFerade

第7章 規制環境

  • 遺伝子治療と規制環境
    • 米国
    • 欧州
    • 日本

第8章 競争環境

  • 主な研究開発(R&D)企業のプロファイル
    • ReGenX Biosciences
    • Oxford BioMedica plc
    • EGEN
    • Sangamo BioSciences
    • BioSante Pharmaceuticals
    • Amsterdam Molecular Therapeutics (AMT)
  • 近年の資本取引(全9件)

第9章 付録

図表一覧

目次

Summary

GBI Research's new report, "Gene Therapy Market to 2018 - Product Development Slowed by Clinical Failures, Close Regulatory Surveillance and High Compliance Standards", provides in-depth analysis of the unmet needs, drivers and barriers that impact the global gene therapy market. The report analyzes technological aspects, advantages and disadvantages of various vectors used in gene therapy, and clinical development trends in gene therapy. It includes in depth analysis of the pipeline molecules, clinical development stages, promising pipeline molecules and therapy areas focused on in R&D. Furthermore, the report provides competitive benchmarking for the leading companies, and analyzes the mergers, acquisitions and licensing agreements that shape the global markets.

This report is built using data and information sourced from proprietary databases, primary and secondary research, and in-house analysis by GBI Research's team of industry experts.

Gene therapies are biological compounds, which modify or replace disease-causing genes. These therapies are the new therapeutic class aimed at treating diseases associated with genetic mutations. Gene therapy promises to provide new treatments for a large number of inherited and acquired diseases. The basic concept of gene therapy is to introduce a piece of genetic material into target cells, which will result in either a cure for the disease or a slowdown in the progression of the disease. It involves the transfer of a functional gene copy into specific cells of an individual in order to repair a faulty gene copy. It may be used to replace a defective gene, or to introduce a new gene to cure a condition.

For example, mutations in genes on the X chromosome lead to X chromosome-linked genetic diseases such as Duchenne muscular dystrophy and hemophilia. Since males have only one copy of the genes from this chromosome, there is no other normal copy available to fulfill a defective gene's function which is present on the X chromosome. If the normal copy of the mutated gene is delivered in the nucleus externally through a delivery agent, the cells can produce the normal gene products and the disease would be treated.

From a commercial perspective, there is a huge unmet need in oncology and autoimmune diseases, amongst others, that could further drive growth of the pharmaceutical and biotech industry. The unmet need is largely driven by the lack of efficacious and safe therapeutic products based on conventional pharmaceutical and biotech research. Gene therapy is a new therapeutic category that has the potential to satisfy this unmet need, especially considering how efficacious and safe this therapeutic category is expected to be.

GBI Research's analysis suggests that therapies developed using gene therapy technology can address the majority of the unmet needs prevailing in the current pharmaceutical market. The inherent structure of gene therapies and their potential to replace the functions of defective genes make them highly effective to knockdown any gene that was previously unapproachable by conventional therapies. Gene therapies are poised to become the next most promising class of drugs in the pharmaceutical industry. Currently there are only three approved products, namely Gendicine, Oncorine and Rexin-G, with a collective market little above $2.8m. Since first movers always have the competitive edge, many large pharmaceutical and biotechnology companies have already commenced their R&D activities on gene therapies.

This report provides insights into the major unmet needs prevailing in the current pharmaceutical industry, and points to gene therapies as the solution to these unmet needs. The report also elucidates the promising late-stage gene therapy pipeline, and provides insights into the gene therapeutics R&D pipeline and funding opportunities.

Scope

  • Annualized various technologies and vectors used in gene therapy.
  • Analysis of the leading therapeutic segments for which clinical development in gene therapy is being conducted.
  • Key drivers and barriers that have a significant impact on the market.
  • Coverage of pipeline molecules in various phases of drug development.
  • Competitive landscape of leading companies. The key companies studied in this report are ReGenX Biosciences, Oxford BioMedica, EGEN, Inc, Sangamo BioSciences, BioSante Pharmaceuticals and Amsterdam Molecular Therapeutics.
  • Key M&A activities and licensing agreements that have taken place between 2010 and 2011 in the global gene therapy market.

Reasons to buy

  • Align your product portfolio to the markets with high growth potential.
  • Develop market-entry and market expansion strategies by identifying the leading therapeutic segments and geographic markets poised for strong growth.
  • Reinforce R&D pipelines by identifying new target mechanisms which can produce first-in-class molecules with greater efficiency and better safety.
  • Develop key strategic initiatives by understanding the key focus areas of leading companies.
  • Exploit in-licensing and out-licensing opportunities by identifying products that could fill portfolio gaps.

Executive Summary

Gene Therapy: the Next Big Step in Cancer Treatments.

The fight against cancer is leading a new movement in gene therapy, as the failure of conventional cancer therapies is fuelling demand for new treatments, according to a new report by healthcare experts GBI Research.

The new report* states that gene therapy technology is still in its nascent stage, and high levels of regulatory surveillance in clinical development is affecting progress. However, the increasing potential of upcoming treatments and shortcomings in traditional therapies is gradually leading to broader acceptance of gene therapy in medicine.

Therapies such as chemotherapy and hormone therapy control the progression of diseases, but are often associated with severe side effects, such as nausea, hair loss and abnormal blood cell counts. Once administered, the drugs induce systemic action throughout the body, and patients often die due to the side effects of treatment rather than the cancer itself. The inability of these conventional therapies to cure diseases has created a significant unmet need in the treatment of cancer, as well as Human Immunodeficiency Virus (HIV), autoimmune diseases, and viral infections.

Targeted therapies such as monoclonal antibodies, stem cell therapies, Ribonucliec Acid (RNA) therapies and gene therapies have initially shown better efficacy and safety profiles compared to chemotherapies.

Gene therapy has several promising drug candidates, which are likely to drive the growth of the gene therapy market if clinical trials are successful. Collategene by AnGes MG, Cardium Therapeutics' Generx, and Vical Incorporation's Allovectin-7 are in development for a wide range of cancer indications, and are expected to compete in the oncology therapeutics market as the market acceptance of gene therapy improves over time.

Gene therapy holds the potential to deliver tumor suppressor genes to an individual, preventing the growth of malignant tumors and reducing metastatic disease. The prevalence of cancer means that 38% of all ongoing gene therapy pipeline projects are for cancer, with particular focus given to rare tumor types such as pancreatic cancer, and highly prevalent tumor types such as breast cancer and prostate cancer, in which case advanced forms of the disease are likely to be the primary targets.

However, patients may have a while to wait. Although the gene therapy pipeline portfolio has 145 pipeline projects under research and development, only 10 projects are in Phase III clinical studies, and only three gene therapy products have reached the market so far.

A Market Authorization Application (MAA) was filed for Glybera and Cerepro, but the Medicines and Healthcare products Regulatory Agency (MHRA) denied their marketing authorization due to a lack of reliable evidence to support the drugs clinical benefit.

Gene Therapy Market to 2018 - Product Development Slowed by Clinical Failures, Close Regulatory Surveillance and High Compliance Standards

This report provides in-depth analysis of the unmet needs, drivers and barriers that impact the global gene therapy market. It analyzes technological aspects, advantages and disadvantages of various vectors used in gene therapy, clinical development trends in gene therapy. In depth analysis is given for the pipeline molecules, clinical development stages, promising pipeline molecules and therapy areas focused in R&D. Furthermore, the report provides competitive benchmarking for the leading companies and analyzes the mergers, acquisitions and licensing agreements that shape the global markets.

This report is built using data and information sourced from proprietary databases, primary and secondary research and in-house analysis by GBI Research's team of industry experts.

Table of Contents

1. Table of Contents

  • 1.1. List of Tables
  • 1.2. List of Figures

2. Introduction

3. Gene Therapy Market - Technology Overview

  • 3.1. Introduction
    • 3.1.1. Gene
    • 3.1.2. Genome
    • 3.1.3. DNA
  • 3.2. Genetic Disorders
  • 3.3. Gene Therapy
    • 3.3.1. Types of Gene Therapies
  • 3.4. Need for Gene Therapy
  • 3.5. Vectors Used in Gene Therapy
    • 3.5.1. Viral Vectors
    • 3.5.2. Non-Viral Vectors
  • 3.6. Role of Gene Therapy in the Pharmaceutical Industry
    • 3.6.1. Upstream Action of Gene Therapies Makes Them More Effective Than the Existing Biologic Therapies

4. Gene Therapy Market - Clinical Development Trends

  • 4.1. Gene Therapy Clinical Development by Year
  • 4.2. Gene Therapy Clinical Development by Vectors Used
  • 4.3. Gene Therapy Clinical Development by Geography
  • 4.4. Gene Therapy Clinical Development by Therapy Area

5. Gene Therapy Market - Market Environment Characterization

  • 5.1. Market Drivers
    • 5.1.1. Failure of Conventional Therapies to Treat Cancers and Other Indications has Created High Unmet Needs
    • 5.1.2. Entry of Gene Therapies in Developed Nations Will Poise the Market Growth
    • 5.1.3. Rising Incidence and Prevalence of Chronic and Life Threatening Diseases
    • 5.1.4. Possible Treatment for Monogenic Conditions Can Give Access to Previously Undeveloped Markets
  • 5.2. Market Restraints
    • 5.2.1. Stringent Regulatory Laws Delay Gene Therapy Development
    • 5.2.2. Safety Concerns
    • 5.2.3. Technical Limitations in Present Technology
  • 5.3. Market Analysis
    • 5.3.1. Strengths
    • 5.3.2. Weakness
    • 5.3.3. Opportunities
    • 5.3.4. Threats
  • 5.4. Current Marketed Products
    • 5.4.1. Gendicine
    • 5.4.2. Oncorine
    • 5.4.3. Rexin-G

6. Gene Therapy Market - Pipeline Analysis

  • 6.1. Research and Development Analysis
    • 6.1.1. R&D Pipeline by Phase
    • 6.1.2. R&D Pipeline by Therapy Area
  • 6.2. Promising Molecules in the Pipeline
    • 6.2.1. Collategene
    • 6.2.2. Allovectin-7
    • 6.2.3. Generx
  • 6.3. Failed Late Stage Products
    • 6.3.1. Glybera
    • 6.3.2. Cerepro
    • 6.3.3. NV1FGF
    • 6.3.4. TNFerade

7. Gene Therapy Market - Regulatory Landscape

  • 7.1. Gene Therapy and the Regulatory Environment
    • 7.1.1. The US
    • 7.1.2. Europe
    • 7.1.3. Japan

8. Gene Therapy Market - Competetive Landscape

  • 8.1. Profiles of Major Companies in Gene Therapy R&D
    • 8.1.1. ReGenX Biosciences
    • 8.1.2. Oxford BioMedica plc
    • 8.1.3. EGEN
    • 8.1.4. Sangamo BioSciences
    • 8.1.5. BioSante Pharmaceuticals
    • 8.1.6. Amsterdam Molecular Therapeutics (AMT)
  • 8.2. Recent Deals in the Gene Therapy Market
    • 8.2.1. MolMed Enters into an Agreement with Fondazione Telethon
    • 8.2.2. GlaxoSmithKline Enters into Co-Development Agreement with McGill University
    • 8.2.3. Amsterdam Molecular Therapeutics Enters into an Agreement with Institut Pasteur and the Consortium
    • 8.2.4. Oxford BioMedica Enters into Research Agreement with Mayo Clinic
    • 8.2.5. RetroSense Therapeutics Enters into Licensing Agreement with Wayne State University
    • 8.2.6. Amsterdam Molecular Expands Licensing Agreement with National Institute of Health
    • 8.2.7. GlaxoSmithKline Enters into Licensing Agreement with Fondazione Telethon
    • 8.2.8. GlaxoSmithKline Enters into Strategic Alliance with Fondazione Telethon and Fondazione San Raffaele
    • 8.2.9. Bluebird bio Enters into Licensing Agreement with INSERM

9. Gene Therapy Market - Appendix

  • 9.1. Abbreviations
  • 9.2. Market Definitions
  • 9.3. Research Methodology
    • 9.3.1. Coverage
    • 9.3.2. Secondary Research
    • 9.3.3. Primary Research
    • 9.3.4. Expert Panel Validation
    • 9.3.5. Section-wise Research Methodology
  • 9.4. Sources
  • 9.5. Contact Us
  • 9.6. Disclaimer

List of Tables

  • Table 1: Gene Therapy Market to 2018, Global, Gene Therapy Clinical Trials by Year, 1989-2011
  • Table 2: Gene Therapy Market to 2018, Global, Gene Therapy Clinical Trials by Type of Vector Used, 1989-2011
  • Table 3: Gene Therapy Market to 2018, Global, Gene Therapy Clinical Trials by Geography, 1989-2011
  • Table 4: Gene Therapy Market to 2018, Global, Gene Therapy Clinical Trials by Therapy Area, 1989-2011
  • Table 5: Gene Therapy Market to 2018, Gendicine Phase II/III Clinical Trials Results, 2005
  • Table 6: Gene Therapy Market to 2018, Global, Pipeline Projects by Phase, %, 2011
  • Table 7: Gene Therapy Market to 2018, Global, Phase III Pipeline Molecules, 2011
  • Table 8: Gene Therapy Market to 2018, Global, Phase II Pipeline Molecules, 2011
  • Table 9: Gene Therapy Market to 2018, Global, Phase I Pipeline Molecules, 2011
  • Table 10: Gene Therapy Market to 2018, Global, IND Filed Pipeline Molecules, 2011
  • Table 11: Gene Therapy Market to 2018, Global, Preclinical Pipeline Molecules, 2011
  • Table 12: Gene Therapy Market to 2018, Global, Discovery Pipeline Molecules, 2011
  • Table 13: Gene Therapy Market to 2018, Global, Pipeline Projects by Therapy Area, %, 2011
  • Table 14: Gene Therapy Market to 2018, Global, Oncology Pipeline Projects, 2011
  • Table 15: Gene Therapy Market to 2018, Global, Central Nervous System Pipeline Projects, 2011
  • Table 16: Gene Therapy Market to 2018, Global, Hematological Disorders Pipeline Projects, 2011
  • Table 17: Gene Therapy Market to 2018, Global, Ophthalmology Disorders Pipeline Projects, 2011
  • Table 18: Gene Therapy Market to 2018, Global, Companies with Gene Therapy Projects, 2011

List of Figures

  • Figure 1: Gene Therapy Market to 2018, Global, Gene Therapy Clinical Trials by Year, 1989-2011
  • Figure 2: Gene Therapy Market to 2018, Global, Gene Therapy Clinical Trials by Type of Vector Used, %, 1989-2011
  • Figure 3: Gene Therapy Market to 2018, Global, Gene Therapy Clinical Trials by Geography, %, 1989-2011
  • Figure 4: Gene Therapy Market to 2018, Global, Gene Therapy Clinical Trials by Therapy Area, %, 1989-2011
  • Figure 5: Gene Therapy Market to 2018, Global, Market Drivers and Restraints, 2011
  • Figure 6: Gene Therapy Market to 2018, Global, Cost vs. Unmet Needs Comparison of Key Drug Classes, 2011
  • Figure 7: Gene Therapy Market to 2018, Global, SWOT Analysis
  • Figure 8: Gene Therapy Market to 2018, Global, Pipeline Projects by Stage of Development, %, 2011
  • Figure 9: Gene Therapy Market to 2018, Global, Pipeline Projects by Therapy Area, %, 2011
  • Figure 10: Gene Therapy Market to 2018, Global, Oncology Pipeline Projects by Stage of Development, %, 2011
  • Figure 11: Gene Therapy Market to 2018, Global, Oncology Pipeline Projects by Indication, %, 2011
  • Figure 12: Gene Therapy Market to 2018, Global, Central Nervous System Pipeline Projects by Stage of Development, %, 2011
  • Figure 13: Gene Therapy Market to 2018, Global, Central Nervous System Pipeline Projects by Indication, %, 2011
  • Figure 14: Gene Therapy Market to 2018, Global, Hematological Disorders Pipeline Projects by Stage of Development, %, 2011
  • Figure 15: Gene Therapy Market to 2018, Global, Hematological Disorders Pipeline Projects by Indication, %, 2011
  • Figure 16: Gene Therapy Market to 2018, Global, Ophthalmology Disorders Pipeline Projects by Stage of Development, %, 2011
  • Figure 17: Gene Therapy Market to 2018, Global, Ophthalmology Disorders Pipeline Projects by Indication, %, 2011
  • Figure 18: Gene Therapy Market to 2018, Global, Companies with Gene Therapy Projects in the Pipeline, %, 2011
  • Figure 19: ReGenX Biosciences, SWOT Analysis, 2012
  • Figure 20: Oxford BioMedica, SWOT Analysis, 2012
  • Figure 21: EGEN, SWOT Analysis, 2012
  • Figure 22: Sangamo BioSciences, SWOT Analysis, 2012
  • Figure 23: BioSante Pharmaceuticals, SWOT Analysis, 2012
  • Figure 24: Amsterdam Molecular Therapeutics, SWOT Analysis, 2012
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