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Business Plan Presentation (May 12, 2000)
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Business Plan Presentation (May 12, 2000)


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  • Clinical Trial Partnership Strategy can utilize a multitude of resources and skills to accomplish this trial.
  • Peter Lobel Ph. D - Therapeutic Investigator Robert Wood Johnson Med School 679 Hoes Lane Piscataway, NJ. 08854 Evan Snyder M.D., Ph. D - Therapeutic Investigator Assistant Professor of Neurology - Harvard 300 Longwood Ave. CH- Enders 248 Boston, MA. 02115     William Mobley M.D., Ph. D - Therapeutic Investigator Stanford University School of Medicine Department of Neurology SUMC H3160 Stanford, CA. 94305-5235 David Sleat Ph. D.- Therapeutic Investigator Center for Advanced Biotechnology and Medicine 675 Hoes Lane Piscataway, NJ. 08854     Jonathan Cooper Ph. D - Therapeutic Investigator Stanford University School of Medicine Department of Neurology SUMC H3160 Stanford, CA. 94305-5235 Martin Katz Ph. D. - Therapeutic Investigator University of Missouri One Hospital Drive Columbia, MO. 65212     Sandra L. Hofmann, M.D., Ph. D. - Therapeutic Investigator Southwestern Medical Center Department of Internal Medicine University of Texas 5323 Harry Hines Blvd. Dallas, TX. 75235-8593           The research effort for this clinical trial has a scientific advisory board. Key members of this board is:   Michael Blaese MD - Advisor 300 Pheasant Run Newtown PA. 18940 Rose-Mary Boustany MD - Advisor/Clinician Duke University Medical Center MSRB Box 2604 Research Drive Durham, NC. 27710  
  • CLN1/CLN2 – 39 Million LSDs – 242 Million Metabolic Disorders 2.5 Billion
  • The United States, Canada, Japan, China, Australia, and each country in Europe are included in the revenue forecast projections 50K per treatment is relatively conservative. Compare other life saving procedures (heart transplant….)
  • $39 Million dollar Market
  • Being on the leading edge of science will separate your organization from all the others. We can test mice and monkeys forever but we still will not be able to answer questions about humans. It is time to validate science.
  • Many steps can be done simultaneously
  • Many steps can be done simultaneously
  • Transcript

    • 1. CLN1/CLN2 Gene Therapy Business Plan
    • 2. Objectives
      • Introduce a therapy to CLN1 and CLN2 children for treatment of their disorder.
      • Develop a model for other AAV gene therapy clinical trials. Gaining vital information to be leveraged in future gene transfer applications.
    • 3. Keys to Success
      • Clinical Trial Sponsor
        • Interest in or knowledge of gene therapy principles
        • Commitment and dedication to the successful treatment of NCL patients
        • Previous clinical trial experience
        • Ability to produce or acquire, clinical grade AAV
        • A center committed to host a site for the clinical trial
        • Committed clinicians with experience in degenerative neurological disorders
      • Clinical Trial Partnership Strategy
    • 4. Organizational Structure
      • NCLRA
      • NIH
      • FDA
      • Mark S. Sands
      • Organizations
      • Universities
      • Therapeutic Investigators and Advisors
    • 5. AAV Viability
      • Lysosomal Storage Diseases
        • Enzyme Deficiency
        • Cross Correction
      • Little or no toxicity with high expression
      • MPS VII Results
    • 6. AAV Viability
      • Lysosomal Storage Diseases
      • Little or no toxicity with high expression
      • MPS VII Results
    • 7. Implementation Strategy
      • CLN1 and CLN2
      • Other LSDs
      • Metabolic Disorders
    • 8. Lysosomal Storage Disorders Projections Table
    • 9. Metabolic Disorder Prevalence
    • 10. Metabolic Disorder Projections
    • 11. NCL Prevalence
      • NCLs Prevalence numbers are 1 : 12,000 to 1 : 78,000
      • 1 : 20800 average NCL prevalence
    • 12. CLN1 and CLN2 Prevalence
    • 13. CLN1/CLN2 Financial Plan
      • $31 Million Fifth Year Bottom Line
      • Break-even and Profit in Year 2
      • Total Estimated Start-up Cost are $1.5 Million
        • Obtaining remaining pre-clinical data and protocol development has an estimated cost of $200,000
          • Protocol development ($60k)
          • Finalizing necessary pre-clinical Data ($60k)
          • Performing primate testing ($80k)
        • Estimated Clinical Trial Funding is approximately $1.3 Million
    • 14. Five Year Financial Forecast
    • 15. Profit and Break-Even Chart
    • 16. Why CLN1/CLN2?
      • Prevalence of LSDs (enzyme deficient disorder)
      • Prognosis of CLN1/CLN2 patients
      • NIH Involvement
      • FDA’s Orphan Drug Act – Fast Tracking IND
      • Consenting Patient Population
      • Leading Scientists
      • Promising Financial Returns
      • Establish Gene Therapy Info-structure / Model
      • Answer Unknown Questions by Validating Science
    • 17. Next Steps
      • Establish Clinical Trial Sponsor Partnerships
        • Sponsorship
        • Trial Administration
        • Trial Sites
        • Clinicians and Surgeons
      • Obtain Clinical Grade Vector
      • Gather “Necessary” Pre-Clinical Data
      • Develop Trial Protocol and Points of Consideration (IRB, RAC, FDA)
      • Finalize IND for Submission
    • 18. Next Steps