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CLN1/CLN2 Gene Therapy Business Plan. Objectives l Introduce a therapy to CLN1 and CLN2 children for treatment of their disorder. l Develop a model for.

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Presentation on theme: "CLN1/CLN2 Gene Therapy Business Plan. Objectives l Introduce a therapy to CLN1 and CLN2 children for treatment of their disorder. l Develop a model for."— Presentation transcript:

1 CLN1/CLN2 Gene Therapy Business Plan

2 Objectives l Introduce a therapy to CLN1 and CLN2 children for treatment of their disorder. l 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 l 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 l Clinical Trial Partnership Strategy

4 Organizational Structure l NCLRA l NIH l FDA l Mark S. Sands l Organizations l Universities l Therapeutic Investigators and Advisors

5 AAV Viability l Lysosomal Storage Diseases –Enzyme Deficiency –Cross Correction l Little or no toxicity with high expression l MPS VII Results

6 AAV Viability l Lysosomal Storage Diseases l Little or no toxicity with high expression l MPS VII Results

7 Implementation Strategy l CLN1 and CLN2 l Other LSDs l Metabolic Disorders

8 Lysosomal Storage Disorders Projections Table

9 Metabolic Disorder Prevalence

10 Metabolic Disorder Projections

11 NCL Prevalence l NCLs Prevalence numbers are 1 : 12,000 to 1 : 78,000 l 1 : average NCL prevalence

12 CLN1 and CLN2 Prevalence

13 CLN1/CLN2 Financial Plan l $31 Million Fifth Year Bottom Line l Break-even and Profit in Year 2 l 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? l Prevalence of LSDs (enzyme deficient disorder) l Prognosis of CLN1/CLN2 patients l NIH Involvement l FDAs Orphan Drug Act – Fast Tracking IND l Consenting Patient Population l Leading Scientists l Promising Financial Returns l Establish Gene Therapy Info-structure / Model l Answer Unknown Questions by Validating Science

17 Next Steps l Establish Clinical Trial Sponsor Partnerships –Sponsorship –Trial Administration –Trial Sites –Clinicians and Surgeons l Obtain Clinical Grade Vector l Gather Necessary Pre-Clinical Data l Develop Trial Protocol and Points of Consideration (IRB, RAC, FDA) l Finalize IND for Submission

18 Next Steps


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