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Novel Therapies for Rare and Genetic Diseases

  • ID: 4591038
  • Report
  • June 2018
  • Region: Global
  • 92 Pages
  • Frost & Sullivan
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Unveiling One of the Most Promising Fields of Therapeutic Technology Synergy

Recent advancement in the development of novel platforms of smart targeting small molecules, gene therapy, stem cell therapy, gene modification, and gene rectification technologies pose new prospects for therapies and cures for rare and genetic diseases. More than 80% of rare diseases are genetic in origin, so that gene and cell therapies are being proposed to treat them. Moreover, human stem cell therapy is suggested as one of the most promising alternatives. Furthermore, genetically modified stem cells and autologous induced pluripotent stem cells, synergistically coalesced with zinc-finger proteins and transactivator-like effector nucleases, are targeted as the selection of choice for the ex vivo repair of specific mutated deoxyribonucleic acid (DNA) sequences.

Similarly, patient specific induced pluripotent stem cells can be edited and shifted back into the patient, leading to a new approach to precision medicine. In addition, once homed, stem cells secrete a vast number of paracrine factors that could become new therapeutic tools in the treatment of rare and genetic diseases.

Therefore, next-generation small molecules, gene therapy, stem cell therapy, and gene editing for DNA repair are promising approaches to the treatment of rare and genetic diseases. This research service (RS) depicts the current landscape and the new trends in the development of novel therapies for rare and genetic diseases, focused on highly interrelated and synergistic approaches.

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1. Executive Summary
1.1 Rare and Genetic Diseases: The Bigger Picture
1.2 Research Focus: Advancing New Therapeutics
1.3 Research Scope: Unveiling Novel Therapies
1.4 Analysis Framework: Core Value
1.5 Research Methodology: Five Steps toward Success

2. Technology Snapshot and Trends
2.1 Key Elements of Analysis: The ‘Rare’ Concept
2.2 Brief Overview of Rare Diseases in the Industry
2.3 Emerging Opportunities for Rare Diseases
2.4 Medicines in Development for Rare Diseases

3. Technology Status Review and Assessment
3.1 Technologies Converging for Rare Diseases Treatment
3.2 Small Molecules to Treat Rare Diseases
3.3 Cell Therapies to Treat Rare Diseases
3.4 DNA-based Therapies to Treat Rare Diseases
3.5 RNA-based Therapies to Treat Rare Diseases
3.6 Gene Editing Technologies to Treat Rare Diseases
3.7 New Approaches Driving Rare Disease Solutions
3.8 Technology Synergy Reshaping Value Chain
3.9 Technology and Business Landscape: Accelerators
3.10 Technology and Business Landscape: Challenges

4. Impact of Rare Disease Therapies on the Pharmaceutical Industry
4.1 Market Dynamics and Opportunities
4.2 Market Potential: Technology Focus Segmentation
4.3 Market Potential: Therapeutic Area Segmentation
4.4 Market Potential: Regional Approach Segmentation
4.5 Prominent Pharmaceutical Market Leaders
4.6 Regional Highlights for Rare Disease Therapeutics
4.7 Global Programs and Initiatives for Rare Diseases

5. Intellectual Property Analysis
5.1 Breakthrough Innovation Trends
5.2 Technology Transfer Assessment and Perceptions
5.3 Disruptive Small Molecule Technology Discoveries
5.4 Disruptive Cell Therapy Discoveries
5.5 Disruptive DNA-based Therapy Discoveries
5.6 Disruptive RNA-based Therapy Discoveries
5.7 Disruptive Gene Editing Discoveries

6. Funding and Investment Landscape
6.1 Funding and Investment Models and Adoption
6.2 Funding Long Shots, Securitization, and Credit
6.3 Pricing Models and Global Adoption
6.4 Funding and Investment Landscape by Technology
6.5 Small Molecules: Mergers, Acquisitions, Collaborations
6.6 Cell Therapies: Mergers, Acquisitions, Collaborations
6.7 DNA Therapies: Mergers, Acquisitions, Collaborations
6.8 RNA Therapies: Mergers, Acquisitions, Collaborations
6.9 Gene Editing: Mergers, Acquisitions, Collaborations
6.10 Mergers, Acquisitions and Partnerships Snapshot

7. Technology Roadmapping
7.1 Technology Maturity Level and Description
7.2 Small Molecules Technology Maturity
7.3 Small Molecules - Drivers and Restraints
7.4 Cell Therapies - Technology Maturity
7.5 Cell Therapies - Drivers and Restraints
7.6 DNA-based Therapies - Technology Maturity
7.7 DNA-based Therapies - Drivers and Restraints
7.8 RNA-based Therapies Technology Maturity
7.9 RNA-based Therapies - Drivers and Restraints
7.10 Gene Editing Technology Maturity
7.11 Gene Editing Technologies - Drivers and Restraints
7.12 Roadmap Tapping into Technology Synergy
7.13 Companies Succeeding in the Rare Disease Space
7.14 The Road Ahead: The P6 Innovation Model
7.15 Business Model Hybridization
7.16 Overall Environmental Impact Factors
7.17 Future Perspective for Rare Disease Therapeutics

8. Performance Benchmarking and Strategic Recommendations
8.1 Multicriteria Approach for Benchmarking
8.2 Strategic Portfolio Assessment
8.3 Level 1 Criteria: Innovation Development
8.4 Level 1 Criteria: Financial Performance
8.5 Level 1 Criteria: Market Evolution
8.6 Level 2 Criteria: Key Actions for Success
8.7 Analytic Performance Results
8.8 Multicriteria Final Assessment
8.9 Rare Disease Therapy Platforms to Watch
8.10 Strategic Business Cases #1-4
8.11 Strategic Business Cases #5-8
8.12 Strategic Business Cases #9-12
8.13 Strategic Imperatives: Critical Success Factors

9. Key Industry Influencers
9.1 Key Industry Influencers
9.2 Legal Disclaimer

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