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General "Boiler Plate" Grant Proposal #2
General "Boiler Plate" Grant Proposal #2
General "Boiler Plate" Grant Proposal #2
General "Boiler Plate" Grant Proposal #2
General "Boiler Plate" Grant Proposal #2
General "Boiler Plate" Grant Proposal #2
General "Boiler Plate" Grant Proposal #2
General "Boiler Plate" Grant Proposal #2
General "Boiler Plate" Grant Proposal #2
General "Boiler Plate" Grant Proposal #2
General "Boiler Plate" Grant Proposal #2
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General "Boiler Plate" Grant Proposal #2

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  • 1. Nathan’s Battle Grant Request Proposal
  • 2. Introduction The Nathan’s Battle Foundation was established in 1999 and formally received Non-For-Profit determination in May of 2000. Rare genetic diseases afflict hundreds of thousands of children across the world with little hope for therapy. One such disease, Late Infantile Batten disease, afflicted a little boy named Nathan Milto. Late infantile batten disease is a fatal, rare degenerative neurological disease. This disease takes no mercy on its young victims. Children typically have seizures, loss of motor control, as well as the ability to think and reason coherently and end up bedridden requiring a feeding tube for nourishment with the average life expectancy of ten years of age. Nathan’s parents, Phil and Tricia Milto, identified a need to spearhead therapeutic research for this devastating disorder. The Milto’s discovered that very little scientific research was being performed in this area, thus the need for such a foundation. After organizing current researchers and related scientist, the Milto’s uncovered a few viable therapeutic options for Late Infantile Batten disease. The foundation continues to drive the development for these therapies and has been extremely effective in accomplishing its goals. The Nathan’s Battle Foundation’s efforts will aid and advance the development of therapies for numerous rare diseases and has been a leader in propelling medical research for the thousands of afflicted children with rare diseases around the world. Need/Problem/Issue Statement Current project funding request are for developing a gene therapy for Late Infantile Batten disease with Institute of Genetic Medicine Weill Medical College of Cornell University. A formal feasibility study has been performed to professionally confirm the viability of such a therapy and it was the conclusion of Cornell University, that it is possible to initiate a clinical trial for the CNS of Late Infantile Batten disease. A detailed work plan has been developed for this project with tasks, milestones, and timeframes. The objective of this project is to develop gene therapy for the central nervous system for Late Infantile Batten disease. The intention of this therapy is to cure this disease. Once successful, over 40 other diseases will benefit this life saving therapy. The project is scheduled to be completed within thirty months and can be accelerated with additional resources applied.
  • 3. Major milestones are: Timeframe Milestones Min. Estimated Cost 11/12/01 Drug development and testing procedures $300,000 1/21/02 Pre-clinical studies for distribution and $500,000 expression 5/15/02 GMP vector process development $300,000 6/28/02 Clinical protocol development $500,000 2/10/03 Toxicology studies Previously included 6/24/03 FDA submission and approvals Previously included 8/05/03 Clinical trial initiation $500,000 Minimum Estimated Total Costs: $2,100,000 Definitive results will be produced from this project. Return from all funding contributions can be measured via accomplishments of project milestones and the ultimate objective of saving children lives. Expected Impact of Project The development of a gene therapy will directly benefit the over 500 hundred children that are afflicted with this gruesome disease through out the world. The potential of this project can have an impact on over 40 other rare diseases, which would include a population of over 50,000 fatally ill people. The principle of this project is to pioneer rare disease therapy development to create a platform for other rare diseases. Budget The current total estimated budget for the LINCL gene therapy project is $2.1 million dollars. The project costs are derived from the project work plan prepared by the Institute of Genetic Medicine Weill Medical College of Cornell University (see appendix for copy of work plan). The work plan specifies tasks and milestones associated with the project. These tasks will require professional resources and associated supplies to develop the drug. These funds will be used to accomplish major project milestone, including the first phase of a life saving clinical trial on humans. The major project milestones involve: the manufacturing of the drug, remaining necessary pre-clinical testing, clinical protocol development, regulatory approval process, and human administration in
  • 4. the first phase of the clinical trial. The costs associated with each milestone are inclusive of scientific materials, laboratory specimens, equipment, professional services, and indirect cost. Cornell University charges a reduced rate for indirect charges for Non-For-Profit organizations of 25%. The Nathan’s Battle Foundation will contribute all personnel time at no charge to the project. Copies of the project budget from the first two stages of the LINCL project are included in the appendix. Budgeted Project Milestone Expenses Amount Vector and analytic development $300,000 Initial vector construction Develop analytical methods Pre-clinical Studies $500,000 Distribution Studies Expression Studies Toxicology studies GMP clinical grade vector process development $300,000 GMP Process development GMP characterization GMP batch development GMP clinical production and quality control Clinical protocol development $500,000 Clinical protocol development Assemble clinical team IRB submission RAC submission FDA submission Phase I Human Clinical Trial $500,000 Commence clinical protocol Total estimated expenses $2,100,000 2001 Project Budget: Percentage Amount Milestone completed in ‘01 Needed Vector and analytic 100% of $300,000 $300,000 development Pre-clinical Studies 80% of $500,000 $400,000 GMP clinical grade 25% of $300,000 $75,000 vector process development Clinical protocol 25%of $500,000 $125,000 development
  • 5. Phase I Human Clinical 0% of $500,000 $0 Trial Total estimated $900,000 expenses for 2001 2002 Project Budget: Percentage Amount Milestone completed in ‘02 Needed Vector and analytic 100% of $300,000 $0 development Pre-clinical Studies 20% of $500,000 $100,000 GMP clinical grade 75% of $300,000 $225,000 vector process development Clinical protocol 75%of $500,000 $375,000 development Phase I Human Clinical 0% of $500,000 $0 Trial Total estimated $700,000 expenses for 2002 2003 Project Budget: Percentage Amount Milestone completed in ‘03 Needed Vector and analytic 100% of $300,000 $0 development Pre-clinical Studies 100% of $500,000 $0 GMP clinical grade 100% of $300,000 $0 vector process development Clinical protocol 100%of $500,000 $0 development Phase I Human Clinical 100% of $500,000 $500,000 Trial Total estimated $500,000 expenses for 2003
  • 6. Budget Explanation The project costs are associated with the milestones defined in the work plan. These expenses are supported from actual cost of salaries, supplies, laboratory specimens, equipment, and production materials. These cost are in-line with the CLN2 AAV Gene Therapy Business Plan, which was developed in March of 2000. This business plan was reviewed by a number of major biotech companies, such as Genzyme, Avigen, Targeted Genetics, and Cell Genesys. The consensus from these companies was that the expenses would be low for their organizations to perform this work, but an independent Non-For-Profit foundation could accomplish the necessary tasks near the defined costs. A large company has more overhead and bureaucracy to involve in a project of this nature. The business plan was presented at a conference at the NIH in May of 2000 to these major biotech companies (Clinical Trial Initiative Conference Summary is available upon request). The required funding of $2 million is justified from the necessary supplies, resources, personnel, and testing. See appendix for AAV Gene Therapy Business Plan Nathan’s Battle Financial Information Financial Statement Information The Nathan’s Battle Foundation was formally incorporated on 3/27/00 and received the formal letter of 501(c)(3) determination in May of 2000. The Foundation does not currently have a formal financial statement prepared from our CPA firm, but one is currently being prepared to comply with the Non-For- Profit filing dates. In summary given the currently planned fund raising events, the Nathan’s Battle Foundation needs to raise over $652,000 to support the therapy development projects for the 2001 fiscal year. For the fiscal year 2002 given the forecasted event revenue, the foundation needs to raise an additional $605,500 to fund therapy development projects. This funding is necessary to continue the development of therapies for LINCL. Receiving all of the necessary funding in Q3 of 2001 is imperative and for 2002 by Q2. The earlier in the year is important to the viability of the project, without timely funding the project will stop. Total additional funding needed for the next two years of 2001 and 2002, given planned and forecasted events, is $1,257,500. The 2003’s forecasted required funding amount needed for the clinical trial initiation stage of the project is expected to be $500,000. Estimated total funding needed is $1,757,000. The project could be accelerated if we were able to secure the total $1,757,000 up front. Having the funding now would enable us to perform more project tasks in parallel because of the additional resources available to perform the work sooner. The critical path of
  • 7. the project is less than 18 months to the clinic but with restricted funding resources the project will take up to 30 months.. If we can secure the total funding in 2001 the project could be shortened and kids could receive therapy more quickly. The revenues and expenses are as follows: 1999 Revenues and Expenses 1999 Revenue Amount The Nathan’s Battle Benefit Dinner $91,263.75 The Nathan’s Battle Golf outing $16,969 Misc. Donations $4,528.51 Interest Paid $1,111.19 Total Revenue $113,872.49 1999 Expenses The Nathan’s Battle Benefit Dinner $2,083 The Nathan’s Battle Golf outing $3,475.75 NCLRA Research Conference $2,713 Total 1999 Expenses $8,271.75 2000 Revenues and Expenses 2000 Revenue Amount Ringling Brothers and Barnum Bailey $95,000 Circus – Nathan’s Battle Under the Big Top The Nathan’s Battle Golf outing $10,500 Jeff and Teri Stoops sale of car $16,500 donated Grants and Awards – Ruth Lilly $5,000 Misc. Donations $15,000 Interest Paid $1,800 Total Revenue $143,800 2000 Expenses Washington University CLN2 Gene $20,000 Therapy Research – Dr. Sands’s Lab The Nathan’s Battle Under the Big $2,583 Top The Nathan’s Battle Golf outing $2,075.75 NCLRA’s Clinical Trial Initiative $3,000 Conference – May 2000
  • 8. Cornell Feasibility Study $125,000 Total 2000 Expenses $152,658.75 2001 Revenues and Expenses Forecast 2001 Forecasted Revenues Amount The Natalie Fund for Childhood $130,000 Brain Disease Research – CA. The Bobbie Shone Trust – England* $20,000 The Nathan’s Battle Golf outings* $60,500 Grants and Awards* $5,000 Misc. Donations $45,000 Run Walk Event $22,000 Benefit Concert $23,000 Dinner Benefit at the Ritz* $50,000 Interest Paid $4,500 Philanthropic or Future Grant (?) Support* Total Revenue $360,000 + (?) 2001 Forecasted Expenses Cornell Clinical Trial Project $900,000 Dinner Benefit at the Ritz expenses* $3,000 The Nathan’s Battle Golf outings* $4,000 Misc. event expenses $5,000 High Through Put CLN2 Drug $100,000 Screening* Total 2001 Expenses $1,012,000 * These line items are forecasted revenues or expenses that have not actually been received or incurred. The 2001 amount needed to raise including currently planned forecasted events is $652,000 ($1,012,000 - $360,000 = $652,000). 2002 Revenues and Expenses Forecast 2002 Forecasted Revenues Amount The Natalie Fund for Childhood $60,000 Brain Disease Research – CA. The Nathan’s Battle Golf outings $50,000 Grants and Awards $5,000
  • 9. Misc. Donations $30,000 Interest Paid $4,500 Philanthropic or Future Grant (?) Support Total Revenue $149,500 + (?) 2002 Forecasted Expenses Cornell Clinical Trial Project $700,000 Misc. event expenses $5,000 High Through Put CLN2 Drug $50,000 Screening Total 2002 Expenses $755,000 The 2002 amount needed to raise including currently planned forecasted events is $605,500 (755,000 - $149,500 = $605,500). Total additional funding needed for the next two years of 2001 and 2002, given planned and forecasted events, is $1,257,500. The 2003’s forecasted required funding needed for the clinical trial initiation stage of the project is expected to be near $500,000. Estimated total funding needed is $1,700,000. Balance Sheet Information 1999 Assets and Liabilities 1999 Assets Amount Cash $105,540.74 Other Assets $0 Total Assets $105,540.74 1999 Liabilities Outstanding Debt $0 Total 1999 Liabilities $0 2000 Assets and Liabilities 2000 Assets Amount Cash $96,681.99 Other Assets $0 Total Assets $96,681.99
  • 10. 2000 Liabilities Outstanding Debt $0 Total 2000 Liabilities $0 Staff and Facilities The Institute of Genetic Medicine Weill Medical College of Cornell University will perform the Gene Therapy project for Late Infantile Batten Disease. The Weill Medical College has a state of the art facility that was newly renovated in 1999 to make it a world-class facility. This facility is one of the only laboratories that have the capability of manufacturing clinical grade AAV GMP vector. The Institute of Genetic Medicine at the Weill Medical College of Cornell University is lead by Dr. Ronald Crystal. Dr. Ronal Crystal is a world renown leader in the gene therapy field. Dr. Crytsal has taken potential therapies to the FDA and has experience executing and performing clinical trials. Dr. Crystal’s leadership of this project ensures the success of this extremely promising project. The excitement in the scientific community regarding this project will foster the necessary collaborations to provide the nations best experts to give guidance and direction. The project will be managed by a partnership between the Cornell Institute of Genetic Medicine and Nathan’s Battle Foundation. This partnership ensures the success of the trial based on the main focus of each party involved. The Nathan’s Battle Foundation will ensure that the project will be focused and progresses to accomplish milestones on an aggressive schedule. The NBF will utilize its managerial skills and developed relationships to aid in propelling the project to the clinic. Evaluation Method The gene therapy project can be easily evaluated by reviewing the progress against the tasks and time frames defined in the work plan. Following the work plan timeline closely will be a major emphasis. We are fighting time with this disease so the project will be measured regularly to facilitate the timely success.
  • 11. Continuation or Sustainability Strategies Once the project is completed and the first phases of a clinical trial are completed, we will have proven the efficacy of the drug for a biotech company to manufacture. Negotiations with a biotech company (Genzyme) have been established to commercialize the drug to make it available to the community (see the attached Non-For-Profit Therapy Development Model Diagram). Appendices AAV Gene Therapy Business Plan Cornell Project Feasibility Study Proposal for stages 1 and 2 Cornell Feasibility Study cover letter Cornell Project Work Plan Non-For-Profit Therapy Development Model Diagram Feasibility Study of Gene Therapy of Late Infantile Neuronal Ceroid Lipofuscinosis (available upon request 56 pages) Clinical Trial Initiative Conference Summary (available upon request 51 pages)

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