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Gene Therapy: Moving Toward Commercialization

Gene Therapy: Moving Toward Commercialization

Cover-imageInsight 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


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
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

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