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Gene Editing and Gene Therapy Breakthroughs Propelling Mainstream Treatments

Gene Editing and Gene Therapy Breakthroughs Propelling Mainstream Treatments

The drug approvals of Yescarta (axicabtagene ciloleucel), Luxturna (Voretigene neparvovec) and Kymriah (Tisagenlecleucel) have turned a leaf in the pharmaceutical chapter of gene therapy development. Currently, genetic disorders do not have satisfactory treatment outcomes. This is in part due to the inability of small molecule drugs and enzyme replacement therapies, usually prescribed for inherited diseases, to correct the root cause of the disorders. In other words, they are not corrective therapies. Gene therapy offers exactly what small molecule drugs and enzyme therapy therapies don't- they can offer corrective solutions for inherited disorders.

A new wave of gene therapy breakthroughs can be expected to follow in the next few years after a two-decade long hiatus that witnessed very few proven gene therapy drug candidates. This potential in-flux of gene therapy candidates is due to the two fundamental factors. First, the refining and development of cutting-edge vector platform both by academia and by pharmaceutical industry. This has resulted in the widely used advanced Adeno-associated viral vectors and Lentiviral vectors, both of which are used according to the applicability. Second, the development of novel gene editing tools such as TALENs, Meganucleases, CRISPR/Cas along with the improvement of old techniques such as ZFNs have helped improve the translational capability of gene therapies currently in clinical development.

In the following study, the development of gene editing tools as well as gene-transfer enabling vectors is traced over the last decade or so while also putting into perspective the number of gene therapy candidates currently in clinical development in addition to the various potential cures and satisfactory treatments that await patients affected by genetic diseases .


  • Executive Summary
    • Research Scope - Application of Technology
    • Research Methodology - The Frost & Sullivan Core Value
    • Key Findings
  • Technology Snapshot
    • Currently Available Gene Therapy Treatments on the Market
  • Gene Editing Tools
    • An Introduction to Gene Editing Tools
    • Clustered Regularly Interspaced Short Palindromic Repeats
      • CRISPR Patent Battle- Event Timeline
    • Zinc Finger Nucleases (ZFNs)
    • Transcription Activator-like Effector Nucleases
    • ARC- Nucleases (ARCUS)
  • Gene Therapy Platform
    • Building a Gene Therapy Platform- Technology Segmentation
    • Methods of Gene Therapy and types of vectors used
    • A Timeline Highlighting Gene Therapy Breakthroughs
    • More Recent Successes in Gene Therapy Development
    • A Brief Account of Setbacks Encountered in Gene Therapy Development Over the Years
    • Viral Vectors- Overview
  • Drivers and Challenges of Gene Therapy Technology
    • Key Factors Aiding Development of Gene Therapy Breakthroughs
    • Key Factors Restricting the Growth of Gene Therapy Technology
  • Major Companies
    • A Comprehensive List of Gene Therapy Companies (US & EU)
  • Drug Development Pipeline of Potential Breakthrough Gene Therapies
    • Gene Therapy Leads in Pre-clinical Development
    • Gene Therapies in Phase 1 Clinical Study
    • Gene Therapies in Phase 2 Clinical Study
    • Gene Therapies in Phase 3 Clinical Development
    • Gene Therapies Segmentation Based on Development phase
  • Intellectual Property Landscape of Gene Editing and Gene Therapy Technology
    • Patent Research Scope and Concepts
    • Top 20 Patent Holders in the Pharmaceutical Industry
      • Table Top 20 Patent Holders, Global, 2008-2018*
    • Global Office-wise Patent Distribution Trend- Top 3 countries
      • Table Organ-on-a-chip Patent Portfolio Distribution by Patent Offices, Global, 2008-2018*
    • Patent Trends of Gene Editing Tools, 2015-2017
    • Year-wise Patent Publication Related to Gene Therapy, 2010-2018
      • Table Publication Year-wise Distribution of Gene Therapy-related Patents, Global, 2010-2018*
  • Growth Opportunities
    • Growth Opportunity 1: CNS and Heart Diseases
    • Growth Opportunity 2: One-time Treatment Design
    • Growth Opportunity 3: Better Pricing Model
  • Appendix
    • Key Contacts

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