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Business Plan Presentation (May 12, 2000)

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Business Plan Presentation (May 12, 2000)

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

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