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Innovative Treatment Options for Restoring Sight: Current Advances and Market Potential

This comprehensive overview focuses on the latest treatment options for restoring sight in Retinitis Pigmentosa (RP) patients, highlighting innovative methods such as retinal prosthesis, optogenetic therapies, and gene therapy. Despite Vitamin A showing inconsistent results, advancements in light-sensitive gene therapy and rod-cone regeneration are promising. The analysis delves into the competitive landscape, estimated market sizes of $600 million in the US, and potential revenue models. It addresses the significant regulatory and technical risks while outlining funding strategies and development timelines essential to bring these therapies to market.

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Innovative Treatment Options for Restoring Sight: Current Advances and Market Potential

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Presentation Transcript

  1. Restoring Sight

  2. Treatment Options Currently available: Vitamin A Has not shown consistent benefits in clinical evaluation In clinical trials: Retinal Prosthesis Vision limited to few pixels

  3. Optogenetic Treatment for RP 1. Algae 2. Gene therapy injection Light-sensitive genes are extracted Genes are added to patient’s DNA 3. Light mediation Light is shone directly into eyes Re-grow rods + cones in retina 5. A Better Life

  4. Competitive Analysis

  5. Patient Population Size Pricing Benchmarks Target market size: 10,000 x$60,000 = $600 Million

  6. Bottom Up Market Size Analysis US target market size: $540 million

  7. Revenue Model Therapeutic Package $$$ Patient Payer Purchaser User

  8. Partners & Allies Intellectual Property Grants $$$ IP $$$ Venture Capital Equity Clinical Trials $$$ Core Competency: Research + Development $$$ Data, FDA Approval $$$ Manufacturers Gene Therapy Serum Optical Device

  9. Key Stakeholders

  10. Timeline Pre-Animal Research Safety & Tolerability Research Animal Research Preclinical Research Phase I Phase II Phase III Clinical Trials Market launch year 3 year 1 year 2 today year 4 year 5

  11. Major Risks Technology Regulatory Regulatory Market Market Market Clinical Development Pre-Clinical

  12. Funding

  13. Major Expenses Patent and Licensing Costs 5 patents * ~40K = ~$200K Development Hiring the Initial Team 3 yrs * (CEO+CSO) = ~1MM 3 yrs * (key hires) = ~1.5MM Trial Contractors $400K (pre-clinical) + $50K (animal) Pre-clinical Research Lab Upkeep 3 yrs * $250K = $750K Clinical Trials $4000/patient * 200 = $800K Clinical Sales / Marketing 50 reps * $50K = 2.5MM

  14. Initial Core Team Founders Scientific Advisory Board

  15. Key Early Hires

  16. Conclusion Mission: Restoring vision to RP patients who currently lack a solution Market: US market $700-800M World market $2.1-2.4B Risk: Significant technical and regulatory risk; partially mitigated by risk benefit profile for RP Future: Platform technology with wide IP coverage, has spectrum of promising therapeutic applications

  17. Acknowledgements

  18. Appendix

  19. Global Market Size

  20. New patient market size US target market size: $14 million

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