Date Published
November 2015

Page Count 
187

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Table of Contents

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