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: Frost & Sullivan’s Core Value

1.5 Research Methodology: Five Steps toward Success

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

3.1 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

4.1 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

5.1 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

6.1 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

7.1 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

8.1 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

9.1 Key Industry Influencers

9.1 Key Industry Influencers (continued)

9.1 Key Industry Influencers (continued)

Legal Disclaimer