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Market Research Report

Gene Therapy: Moving Toward Commercialization

Published by Insight Pharma Reports Product code 344769
Published Content info 147 Pages
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Gene Therapy: Moving Toward Commercialization
Published: November 6, 2015 Content info: 147 Pages
Description

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

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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