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Gene Editing and Gene Therapy Breakthroughs Propelling Mainstream Treatments: 2018 Reearch Report -

The "Gene Editing and Gene Therapy Breakthroughs Propelling Mainstream Treatments" report has been added to's offering.

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.

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.

Key Topics Covered:

1.0 Executive Summary

2.0 Technology Snapshot

3.0 Gene Editing Tools

4.0 Gene Therapy Platform

5.0 Drivers and Challenges of Gene Therapy Technology

6.0 Major Companies

7.0 Drug Development Pipeline of Potential Breakthrough Gene Therapies

8.0 Intellectual Property Landscape of Gene Editing and Gene Therapy Technology

9.0 Growth Opportunities

10.0 Appendix

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Last Updated: 19-Oct-2018