Gene Editing: The Next Breakthrough in Regenerative Medicine

  • ID: 4421391
  • Report
  • 61 pages
  • Datamonitor Healthcare
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Believed to be one of the great biotechnology breakthroughs, gene editing is a powerful tool in pharmaceutical research that could radically change how certain diseases are treated. The precision and rapid-acting fashion of tools such as zinc-finger nucleases, transcription activator-like effector nucleases, and clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR associated protein 9 (Cas9) create untapped opportunity. But gene editing does not come without challenges and controversy, especially when it comes to safety and ethical issues.

Key academic research performed over the past 20 years has led to important discoveries in CRISPR/Cas9 technology, and has also prompted a fierce intellectual property battle over which organization owns the foundational patents. Nevertheless, out of that research has come a wave of start-up companies that are inching toward commercial clinical development with gene editing therapy candidates. Along the way, these biotechs have secured partners, including large pharmaceutical companies, to advance development and eventually commercialization.
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1 EXECUTIVE SUMMARY
  • Gene editing technologies evolve and improve over time
  • Multiple researchers have contributed to key academic discoveries in CRISPR/Cas9
  • The dispute over foundational CRISPR/Cas9 patents carries on
  • Many challenges and controversies remain in advancing CRISPR/Cas9 technology
  • Many players, from small biotechs to Big Pharma, are developing gene editing drug candidates
  • The gene editing pipeline is a small proportion of regenerative medicine therapies, and is dominated by CRISPR/Cas9
2 EVOLUTION OF GENE EDITING TECHNOLOGY
  • Gene editing can radically change how diseases are targeted
  • Gene editing is an emerging modality within the broader regenerative medicine market
  • Zinc-finger nucleases
  • Transcription activator-like effector nucleases
  • CRISPR/Cas9
  • Bibliography
3 ORIGINS OF ACADEMIC RESEARCH IN CRISPR/CAS9
  • Academic research over the last 20 years has helped to advance CRISPR/Cas9 technology toward human therapeutic use
  • Many start-ups have been borne out of CRISPR/Cas9 academic research
  • Bibliography
4 CRISPR/CAS9 PATENT LITIGATION
  • Select key patent filings, issuances, and decisions
  • Patent pools and cross-licensing may help commercial drug developers avoid intellectual property barriers in the future
  • Bibliography
5 CHALLENGES AND CONTROVERSIES WITH GENE EDITING
  • Process challenges exist for gene editing tools
  • Editing human embryos pose ethical concerns
  • Safety issues plague gene editing
  • As is the case with many regenerative medicines, cost of gene editing therapies may be prohibitive
  • Bibliography
6 KEY COMPANIES IN GENE EDITING
  • Key players in gene editing span both biotech and large pharma companies
  • Profiles of select key gene editing companies
  • Through deal-making, large pharmaceutical companies have gotten involved in gene editing
  • Bibliography
7 GENE EDITING PIPELINE TRENDS
  • CRISPR/Cas9 gene editing candidates represent the majority of the gene editing pipeline
  • ZFN therapies are the most advanced by phase
  • Rare diseases lead gene editing therapy areas
  • CRISPR/Cas9 is now being tested in humans
  • Editas and Intellia are on deck to initiating CRISPR/Cas9 Phase I trials
  • Bibliography
8 APPENDIX
  • Scope
  • Methodology
List of Figures
Figure 1: Zinc finger nuclease gene editing tool
Figure 2: Zinc-finger nuclease advantages and disadvantages
Figure 3: Transcription activator-like effector nuclease gene editing tool
Figure 4: Transcription activator-like effector nucleases advantages and disadvantages
Figure 5: CRISPR/Cas9 gene editing tool
Figure 6: CRISPR/Cas9 advantages and disadvantages
Figure 7: Timeline of select key academic research discoveries in CRISPR/Cas9 as it has evolved toward human therapeutic use, 1987-2013
Figure 8: Timeline of select key patent actions in CRISPR/Cas9 technology, 2012-17
Figure 9: Key challenges and controversies with gene editing
Figure 10: Editas Medicine and CRISPR Therapeutics lead the most active gene editing companies
Figure 11: CRISPR/Cas9 technology dominates the gene editing pipeline, preclinical-Phase II*
Figure 12: First-generation ZFN technology leads to more advanced candidates in the gene editing pipeline
Figure 13: Rare diseases are most often targeted by gene editing candidates
Figure 14: Gene editing pipeline volume by indication

List of Tables
Table 1: Overview of gene editing technologies
Table 2: CRISPR/cas9 start-up company academic origins
Table 3: Key gene editing companies
Table 4: Cellectis' gene editing pipeline
Table 5: CRISPR Therapeutics' gene editing pipeline
Table 6: Editas Medicine' gene editing pipeline
Table 7: Intellia Therapeutics' gene editing pipeline
Table 8: Sangamo Therapeutics' gene editing pipeline
Table 9: Key gene editing deals involving large pharmaceutical companies, 2012-17*
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