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Gene Therapy: Moving Towards Commercialization

  • November 2015
  • -
  • Insight Pharma Reports
  • -
  • 187 pages

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

Gene Therapy: Moving Towards Commercialization
Table of Contents
Executive Summary vi
The potential near-term success of ex vivo gene therapies xxii
Outlook for gene therapy xxv
CHAPTER 1:
History of Gene Therapy 31
Early gene therapy studies in academic and government laboratories 32
The death of Jesse Gelsinger and the moratorium on gene therapy development in the United States 34
Gene therapy as a premature technology 34
Most gene therapy clinical studies still take place in academic and government laboratories 36
The scope of this report 37
CHAPTER 2:
Vectors for gene therapy 39
Retroviral vectors 39
Gammaretroviral vectors 39
Lentiviral vectors 42
A recent review of the use of retroviral vectors in gene therapy for primary immunodeficiencies (PIDs) 42
Adeno-associated virus (AAV) vectors 44
AAV strains and vector development 45
Helper-dependent adenovirus vectors 46
Non-viral vectors for gene therapy 47
Conclusions 48
CHAPTER 3:
uniQure, Glybera, and the Beginning ofGene Therapy Commercialization 50
uniQure's technology platform 50
Approval of Glybera 51
Commercialization of Glybera 53
Iterative improvement of AAV vectors 54
uniQure's pipeline and collaborations 56
Corporate development as a factor in uniQure's success 58
Conclusions 58
CHAPTER 4:
Gene Therapy for Ophthalmological Diseases 60
Why gene therapy for retinal diseases? 60
Companies with clinical-stage gene therapies for retinal diseases 62
Spark Therapeutics 62
Human clinical trials of AAV2- hRPE65v2 64
Breakthrough therapy designation for SPK-RPE65 65
SPK-CHM 65
Spark's programs in other gene therapies 66
Spark Therapeutics as a company 66
GenSight Biologics 66
GS010 (rAAV2/2-CMV-ND4) 67
GS030, a preclinical-stage gene therapy for treatment of RP 68
NightstaRx' AAV2-REP1 68
Avalanche Biotech's AVA-101 69
The Avalanche/Regeneron agreement 70
Oxford BioMedica 70
RetinoStat 71
Sanofi/Oxford BioMedica's SAR 422459 (StarGen) and SAR 421869 (UshStat) 71
Applied Genetic Technologies Corp (AGTC) 72
AGTC gene therapy for XLRS (rAAV2tYF-CB-hRS1) 72
Genzyme's AAV-sFLT01 (soluble VEGF-R) for wet AMD 73
Can gene therapy for ophthalmic diseases provide long-term improvement of vision, or does its effects fade with time? 73
Conclusions 74
CHAPTER 5:
Gene Therapy for Other Rare Diseases 75
Hemophilia and gene therapy 75
The Phase 1 Nathwani studies of gene therapy for hemophilia B 77
Companies with clinical-stage hemophilia genetherapy products 78
Baxalta's AskBio009 (BAX 335) 79
Spark Therapeutics' SPK-FIX 80
uniQure/Chiesi's AMT-060 (AAV5-hFIX) 81
Dimension Therapeutics' FIX gene therapy 81
Clinical-stage gene therapies for selected other rare diseases 81
Gene therapy for adenosine deaminase severe combined immunodeficiency syndrome (ADA-SCID) (GSK2696273) 82
Gene therapy for acute intermittent porphyria (AIP) 83
Gene therapies for Sanfilippo syndrome 84
Gene therapy for metachromatic leukodystrophy (MLD) 85
Conclusions 85
Sam Wadsworth Interview April 16, 2015 86
CHAPTER 6:
Gene Therapy for More Common Diseases 90
Introduction 90
Voyager Therapeutics 91
Voyager's product engine 91
Voyager's clinical program 92
Voyager's preclinical portfolio 94
Oxford BioMedica's PD gene therapy program 95
GeneQuine Therapeutics and gene therapy for osteoarthritis 95
GeneQuine's product portfolio 96
Celladon Corporation's gene therapy for heart failure 96
Conclusions 98
CHAPTER 7:
Ex Vivo Gene Therapy 99
bluebird bio 99
bluebird bio's clinical-stage candidates 100
Lenti-D 100
LentiGlobin BB305 101
bluebird's clinical-stage gene therapies—“hot” new company, old technology strategy 103
bluebird's preclinical programs 104
CAR T-cell immunotherapy as an area of ex vivo gene therapy 105
Selected clinical programs in CAR T-cell based immunotherapy 106
Safety issues with CAR T-cell therapies 109
Leading companies and collaborations working on CAR T-cell therapies 110
Conclusions 111
CHAPTER 8:
Gene Editing Technology 112
Editas Medicine 113
Editas' AAV vector-based CRISPR/Cas9 genome editing system 114
Other startup companies pursuing CRISPR/Cas9 genomeediting therapies 116
Sangamo BioSciences, zinc-finger nucleases, and the firstgene-editing clinical studies 116
Sangamo's preclinical pipeline 118
bluebird bio's gene editing programs 118
Conclusions 119
CHAPTER 9:
Summary and Conclusions 121
Chapter 1: History of Gene Therapy 121
Chapter 2: development of improved vectors 123
Chapter 3: uniQure, Glybera, and the beginning of gene therapy commercialization 125
Chapter 4: Gene therapy for ophthalmological diseases 127
Spark Therapeutics 128
GenSight Biologics 129
NightStaRx' AAV2-REP1 130
Avalanche Biotech's AVA-101 130
Oxford Biomedica 131
Applied Genetic Technologies Corp (AGTC) 132
Genzyme's AAV-sFLT01 (soluble VEGF-R) for wet AMD 132
Can gene therapy for ophthalmic diseases provide long-term improvement of vision, or does its effects fade with time? 132
Chapter 5: Gene therapy for other rare diseases 133
Hemophilia and gene therapy 133
The Phase 1 Nathwani studies of gene therapy for hemophilia B 134
Clinical-stage gene therapies for selected other rare diseases 135
Chapter 6: Gene therapy for more common diseases 135
Voyager Therapeutics 136
Oxford BioMedica's Parkinson's disease program 136
GeneQuine Biotherapeutics and gene therapy for osteoarthritis 137
Celladon Corporation's gene therapy for heart failure 138
Outlook for gene therapies for common diseases 138
Chapter 7: Ex vivo gene therapy 139
bluebird bio 139
bluebird bio's clinical-stage candidates 139
Lenti-D 140
LentiGlobin BB305 140
bluebird's clinical-stage gene therapies—“hot” new company, old technology strategy 142
bluebird's preclinical programs 142
CAR T-cell immunotherapy as an area of ex vivo gene therapy 142
Selected clinical programs in CAR T-cell based immunotherapy 143
Safety issues with CAR T-cell therapies 144
Leading companies and collaborations working on CAR T-cell therapies 145
The potential near-term success of ex vivo gene therapies 145
Chapter 8: Gene editing technology 146
Editas' AAV vector-based CRISPR-Cas9 genome editing system 147
Other startup companies pursuing CRISPR/Cas9 genome editing therapies 148
Sangamo BioSciences, zinc-finger nucleases, and the first gene-editing clinical studies 149
Sangamo's preclinical pipeline 149
bluebird bio's gene editing programs 149
Outlook on genome editing technology for gene therapy 150
Insight Pharma Reports survey on gene therapy 151
Outlook for gene therapy 154
Insight Pharma Reports Survey on Gene Therapy (n=88) 157
References 162
About Cambridge Healthtech Institute 183

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