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CLN1/CLN2 Gene Therapy Business Plan

CLN1/CLN2 Gene Therapy Business Plan. 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. Keys to Success.

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CLN1/CLN2 Gene Therapy Business Plan

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  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

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