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市場調查報告書

基因治療:商業化的動向

Gene Therapy: Moving Toward Commercialization

出版商 Insight Pharma Reports 商品編碼 344769
出版日期 內容資訊 英文 147 Pages
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基因治療:商業化的動向 Gene Therapy: Moving Toward Commercialization
出版日期: 2015年11月06日 內容資訊: 英文 147 Pages
簡介

本報告以基因治療是否能從2020年代初到中期取得商業上的成功為重要主題,以商業化為目的之臨床階段的基因治療方案及實行計劃的企業為焦點,提供預計成功率最高的基因治療計劃的類型,及妨礙主要基因治療計劃臨床、商業性成功的障礙等相關分析。

摘要整理

生物外基因治療的短期成功可能性

基因治療的展望

第1章 基因治療的歷史

  • 大學、政府研究所的初期基因治療研究
  • Jesse Gelsinger 少年的死亡和美國的基因治療開發的臨時停止
  • 基因治療是時期尚早的技術
  • 大部分的基因治療臨床研究依然在大學、政府研究所進行
  • 本調查的調查範圍

第2章 基因治療媒介

  • 逆轉錄病毒媒介
  • 伽馬逆轉錄病毒媒介
  • 扳子病毒媒介
  • 原發性免疫缺陷 (PID) 之基因治療的逆轉錄病毒的使用的最新研討
  • 腺病毒相關病毒 (AAV) 媒介
  • AAV株、媒介開發
  • 輔助依賴性腺病毒媒介
  • 基因治療的非病毒載體
  • 結論

第3章 uniQure的Glybera:基因治療商業化的開端

  • uniQure的技術平台
  • Glybera的認證
  • Glybera的商業化
  • AAV媒介的逐次改善
  • uniQure的開發平台、合作
  • uniQure的成功中企業開發是要素
  • 結論

第4章 眼科疾病取向基因治療

  • 為何是視網膜疾病取向的基因治療?
  • 擁有處於臨床階段的視網膜疾病取向基因治療的企業
  • Spark Therapeutics
  • SPK-CHM
  • 企業的Spark Therapeutics
  • GenSight Biologics
  • GS010 (rAAV2/2-CMV-ND4)
  • GS030:RP的治療取向的前臨床階段的基因治療
  • NightstaRx的 AAV2-REP1
  • Avalanche Biotech的AVA-101
  • Oxford BioMedica
  • Applied Genetic Technologies Corp (AGTC)
  • XLRS (rAAV2tYF-CB-hRS1) 之AGTC基因治療
  • 濕型老齡化黃斑部病變取向的Genzyme的 AAV-sFLT01 (soluble VEGF-R)
  • 眼科疾病取向基因治療,能提供視力的長期性的改善嗎,還是其效果會慢慢喪失?
  • 結論

第5章 其他罕見疾病取向基因治療

  • 血友病和基因治療
  • Nathwani 的B型血友病取向基因治療研究的階段I
  • Baxalta的 AskBio009 (BAX 335)
  • Spark Therapeutics的 SPK-FIX
  • uniQure/Chiesi的 AMT-060 (AAV5-hFIX)
  • Dimension Therapeutics的 FIX gene therapy
  • 其他主要罕見疾病的臨床階段的基因治療
  • 對腺苷脫氨酶缺損症複合免疫機能喪失症候群的遺傳基因療法 (ADA-SCID) (GSK2696273)
  • 急性間歇性紫質症 (AIP) 之基因治療
  • 聖菲利柏氏症基因治療
  • 異染性腦白質退化症 (MLD) 之基因治療
  • 結論
  • Sam Wadsworth的採訪

第6章 一般疾病的基因治療

  • 簡介
  • Voyager Therapeutics
  • Oxford BioMedica的PD基因治療計劃
  • GeneQuine Therapeutics 及骨關節炎基因治療
  • Celladon Corporation的心臟衰竭基因治療
  • 結論

第7章 生物外基因治療

  • bluebird bio
  • bluebird bio 臨床階段的候補藥
  • bluebird bio 臨床階段的基因治療,關注的新企業,舊的技術策略
  • bluebird bio 前臨床計劃
  • 生物外基因治療的領域的CAR-T細胞療法 (嵌合抗原受體表現T細胞療法)
  • 結論

第8章 遺傳基因編輯技術

  • Editas Medicine
  • 其他新興企業
  • Sangamo BioSciences、鋅指核酸酶 (ZNF) 、 firstgene-editing 臨床研究
  • Sangamo的前臨床開發平台
  • bluebird bio的遺傳基因編輯計劃
  • 結論

第9章 摘要、結論

參照

關於Cambridge Healthtech Institute

圖表

目錄

Insight Pharma Reports' “Gene Therapy: Moving Toward Commercialization”, outlines the progress of the gene therapy field since its inception in the 1970s, with a special focus on clinical-stage gene therapy programs that are aimed at commercialization, and the companies that are carrying out these programs. A major theme of this report is whether gene therapy can attain commercial success by the early-to-mid 2020s, which types of gene therapy programs have the greatest likelihood of success, and what hurdles might stand in the way of clinical and commercial success of leading gene therapy programs.

In accord with the focus of this report, we have been asking:

  • Whether gene therapy can attain commercial success by the early-to-mid 2020s,
  • Which types of gene therapy programs have the greatest likelihood of success,
  • What hurdles might stand in the way of clinical and commercial success of leading gene therapy programs.

In addition to chapters that focus on various areas of commercial gene therapy, this report includes:

  • An expert interview with Sam Wadsworth, Ph.D., the Chief Scientific Officer of Dimension Therapeutics and former Head of Gene Therapy R&D at Genzyme.
  • Survey data from 88 researchers involved in gene therapy
  • Companies profiled: uniQure, Voyager Therapeutics, Oxford BioMedica, GeneQuine Therapeutics, Celladon Corporation, and bluebird

Topics covered:

  • Development of improved vectors (integrating and non-integrating vectors)
  • Gene therapy for ophthalmological diseases
  • Gene therapy for other rare diseases
  • Clinical-stage gene therapies for selected rare diseases other than hemophilias
  • Gene therapy for more common diseases
  • Companies whose central technology platform involves ex vivo gene therapy
  • CAR T-cell immunotherapy as an area of ex vivo gene therapy
  • Gene editing technology
  • Outlook for gene therapy
  • Market outlook for eight gene therapy products

Table of Contents

Executive Summary

The potential near-term success of ex vivo gene therapies

Outlook for gene therapy

CHAPTER 1:

  • History of Gene Therapy
  • Early gene therapy studies in academic and government laboratories
  • The death of Jesse Gelsinger and the moratorium on gene therapy development in the United States
  • Gene therapy as a premature technology
  • Most gene therapy clinical studies still take place in academic and government laboratories
  • The scope of this report

CHAPTER 2:

  • Vectors for gene therapy
  • Retroviral vectors
  • Gammaretroviral vectors
  • Lentiviral vectors
  • A recent review of the use of retroviral vectors in gene therapy for primary immunodeficiencies (PIDs)
  • Adeno-associated virus (AAV) vectors
  • AAV strains and vector development
  • Helper-dependent adenovirus vectors
  • Non-viral vectors for gene therapy
  • Conclusions

CHAPTER 3:

  • uniQure, Glybera, and the Beginning ofGene Therapy Commercialization
  • uniQure's technology platform
  • Approval of Glybera
  • Commercialization of Glybera
  • Iterative improvement of AAV vectors
  • uniQure's pipeline and collaborations
  • Corporate development as a factor in uniQure's success
  • Conclusions

CHAPTER 4:

  • Gene Therapy for Ophthalmological Diseases
  • Why gene therapy for retinal diseases?
  • Companies with clinical-stage gene therapies for retinal diseases
  • Spark Therapeutics
    • Human clinical trials of AAV2- hRPE65v2
    • Breakthrough therapy designation for SPK-RPE65
  • SPK-CHM
    • Spark's programs in other gene therapies
  • Spark Therapeutics as a company
  • GenSight Biologics
  • GS010 (rAAV2/2-CMV-ND4)
  • GS030, a preclinical-stage gene therapy for treatment of RP
  • NightstaRx' AAV2-REP1
  • Avalanche Biotech's AVA-101
    • The Avalanche/Regeneron agreement
  • Oxford BioMedica
    • RetinoStat
    • Sanofi/Oxford BioMedica's SAR 422459 (StarGen) and SAR 421869 (UshStat)
  • Applied Genetic Technologies Corp (AGTC)
  • AGTC gene therapy for XLRS (rAAV2tYF-CB-hRS1)
  • Genzyme's AAV-sFLT01 (soluble VEGF-R) for wet AMD
  • Can gene therapy for ophthalmic diseases provide long-term improvement of vision, or does its effects fade with time?
  • Conclusions

CHAPTER 5:

  • Gene Therapy for Other Rare Diseases
  • Hemophilia and gene therapy
  • The Phase 1 Nathwani studies of gene therapy for hemophilia B
  • Companies with clinical-stage hemophilia genetherapy products
  • Baxalta's AskBio009 (BAX 335)
  • Spark Therapeutics' SPK-FIX
  • uniQure/Chiesi's AMT-060 (AAV5-hFIX)
  • Dimension Therapeutics' FIX gene therapy
  • Clinical-stage gene therapies for selected other rare diseases
  • Gene therapy for adenosine deaminase severe combined immunodeficiency syndrome (ADA-SCID) (GSK2696273)
  • Gene therapy for acute intermittent porphyria (AIP)
  • Gene therapies for Sanfilippo syndrome
  • Gene therapy for metachromatic leukodystrophy (MLD)
  • Conclusions
  • Sam Wadsworth Interview April 16, 2015

CHAPTER 6:

  • Gene Therapy for More Common Diseases
  • Introduction
  • Voyager Therapeutics
    • Voyager's product engine
    • Voyager's clinical program
    • Voyager's preclinical portfolio
  • Oxford BioMedica's PD gene therapy program
  • GeneQuine Therapeutics and gene therapy for osteoarthritis
    • GeneQuine's product portfolio
  • Celladon Corporation's gene therapy for heart failure
  • Conclusions

CHAPTER 7:

  • Ex Vivo Gene Therapy
  • bluebird bio
  • bluebird bio's clinical-stage candidates
    • Lenti-D
    • LentiGlobin BB305
  • bluebird's clinical-stage gene therapies-“hot” new company, old technology strategy
  • bluebird's preclinical programs
  • CAR T-cell immunotherapy as an area of ex vivo gene therapy
    • Selected clinical programs in CAR T-cell based immunotherapy
    • Safety issues with CAR T-cell therapies
    • Leading companies and collaborations working on CAR T-cell therapies
  • Conclusions

CHAPTER 8:

  • Gene Editing Technology
  • Editas Medicine
    • Editas' AAV vector-based CRISPR/Cas9 genome editing system
  • Other startup companies pursuing CRISPR/Cas9 genomeediting therapies
  • Sangamo BioSciences, zinc-finger nucleases, and the firstgene-editing clinical studies
  • Sangamo's preclinical pipeline
  • bluebird bio's gene editing programs
  • Conclusions

CHAPTER 9:

  • Summary and Conclusions
  • Chapter 1: History of Gene Therapy
  • Chapter 2: development of improved vectors
  • Chapter 3: uniQure, Glybera, and the beginning of gene therapy commercialization
  • Chapter 4: Gene therapy for ophthalmological diseases
  • Spark Therapeutics
  • GenSight Biologics
  • NightStaRx' AAV2-REP1
  • Avalanche Biotech's AVA-101
  • Oxford Biomedica
  • Applied Genetic Technologies Corp (AGTC)
  • Genzyme's AAV-sFLT01 (soluble VEGF-R) for wet AMD
  • Can gene therapy for ophthalmic diseases provide long-term improvement of vision, or does its effects fade with time?
  • Chapter 5: Gene therapy for other rare diseases
  • Hemophilia and gene therapy
  • The Phase 1 Nathwani studies of gene therapy for hemophilia B
  • Clinical-stage gene therapies for selected other rare diseases
  • Chapter 6: Gene therapy for more common diseases
  • Voyager Therapeutics
  • Oxford BioMedica's Parkinson's disease program
  • GeneQuine Biotherapeutics and gene therapy for osteoarthritis
  • Celladon Corporation's gene therapy for heart failure
  • Outlook for gene therapies for common diseases
  • Chapter 7: Ex vivo gene therapy
  • bluebird bio
  • bluebird bio's clinical-stage candidates
    • Lenti-D
    • LentiGlobin BB305
  • bluebird's clinical-stage gene therapies-“hot” new company, old technology strategy
  • bluebird's preclinical programs
  • CAR T-cell immunotherapy as an area of ex vivo gene therapy
    • Selected clinical programs in CAR T-cell based immunotherapy
    • Safety issues with CAR T-cell therapies
    • Leading companies and collaborations working on CAR T-cell therapies
  • The potential near-term success of ex vivo gene therapies
  • Chapter 8: Gene editing technology
    • Editas' AAV vector-based CRISPR-Cas9 genome editing system
  • Other startup companies pursuing CRISPR/Cas9 genome editing therapies
  • Sangamo BioSciences, zinc-finger nucleases, and the first gene-editing clinical studies
  • Sangamo's preclinical pipeline
  • bluebird bio's gene editing programs
  • Outlook on genome editing technology for gene therapy
  • Insight Pharma Reports survey on gene therapy
  • Outlook for gene therapy
  • Insight Pharma Reports Survey on Gene Therapy (n=88)

References

About Cambridge Healthtech Institute

FIGURES

  • Figure 2.1. Gammaretroviral Vector Construction and Packaging 40
  • Figure 3.1. The Glybera Vector DNA 52
  • Figure 4.1. Structure of GenSight AAV2/2-CMV-ND4 Vector (GS010) 68
  • Figure 7.1. CAR T cell 105

TABLES

  • Table 2.1: Leading Gene Therapy Vectors 48
  • Table 3.1. uniQure's Clinical-Stage Pipeline 56
  • Table 4.1: Clinical Stage Gene Therapies for Ophthalmological Diseases 62
  • Table 5.1: Clinical-stage gene therapies for hemophilia B 78
  • Table 5.2: Clinical-Stage Gene Therapies for Selected Other Rare Diseases 82
  • Table 6.1: Voyager's Preclinical Programs 94
  • Table 7.1: bluebird bio's Clinical-Stage Gene Therapies 100
  • Table 7.2: Selected Clinical Programs in CAR T-cell-based Immunotherapy 106
  • Table 8.1: Companies Involved in Gene Editing Technologies 119
  • Table 9.1: Gene therapy products likely to reach the market before 2020 155
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