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Growing Stronger Research Fund Overview Jan 2012 For Donors


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Overview of, a non-profit focused on improving the quality of life of little people through supporting medical research.

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Growing Stronger Research Fund Overview Jan 2012 For Donors

  1. 1. Twins Our mission is to improve the quality of life of little people through supporting medical research Presentation not contain medical advice. This presentation is for January, 2012
  2. 2. SUMMARY Achondroplasia is random and rare People with this condition are also known as little people (LP) or dwarfs People with Achondroplasia typically go through 4-8 surgeries to manage bone related complications Growing Stronger Research Fund 501(c)(3) Goal: Accelerate creation of a drugs to eliminate surgeries Method: Working with leading researchers Status: Currently funding two researchers. Planning to expand to ten research sites.
  3. 3. ACHONDROPLASIA Genetic Mutation 80% of little people are born to average height parents (random, rare) Short limbs forces assistance for common tasks Challenges Short stature (adult ht. ~4ft), spinal compression, bowed legs, sleep apnea Social: Being different. Cannot perform many daily tasks without assistance from stools, extenders, adaptors GREAT NEWS Average intelligence
  4. 4. AWARENESS GROWING 6 seasons of TLC primetime reality show ended in 2010 Current TLC reality show started in 2009 Focused events, groups
  5. 5. AVAILABLE OPTIONS FOR MANAGINGACHONDROPLASIA Lifelong Management of Orthopedic Issues up to 8 surgical procedures to reduce pressure on the upper, middle and lower spine, open air pathways for sleep apnea, straighten bowed legs and make room for crowed teeth. Assistance Devices (ADs) Surgical Limb Lengthening Does not improve health. Painful and dangerous. Only increases height nominally
  6. 6. EMERGING OPPORTUNITYDrug therapy on genes and related proteins makesit possible to manage limited bone growth Responsible Mutant Gene FGFR3 identified in 1994 4p16.3. 90% of cases occur because of a point mutation in one copy. G to A, nucleotide 1138 Potentially reduce the impact of related orthopedic issues Improve quality of life with reduced pain Eliminate need for many assistance devicesNote: There is no cure to correct genetic mutation. However, new therapies can significantly improve the quality of life of little people
  7. 7. Today Potential Achondroplasia RESEARCH TO DRUG TIMELINE therapies 3-5 years 1-3 years 2-5 years Publish lab results Lab IP capture Pre-clinicialDiscovery experiments Companies trials & get interested Live Mouse Models Drug Development Stage Clinical Lab results trials Basic Research and Theory Non-profit funding: For-profit: NIH, University, Foundations Companies, VCs
  8. 8. Today Potential Achondroplasia RESEARCH TO DRUG TIMELINE therapies 3-5 years 1-3 years 2-5 years We need to raise funds to Publish accelerate promising results lab Lab research through theIPlabDiscovery experiments capture Pre-clinicial trials Companies & and into the Live Mouseof drug get interested hands companies Models Drug Development Stage Clinical Lab results trials Basic Research and Theory Non-profit funding: For-profit: NIH, University, Foundations Companies, VCs
  10. 10. ROADMAP FOR RAPID PROGRESS Create scientific advisory board Dr. Michael Ain, Associate Professor, John Hopkins has agreed to join Conduct bi-annual peer advice and drive research with a focused goal Create Relationships with Pharma Create relationships with value added researchers to accelerate export of discoveries to Pharmas and drug development
  11. 11. ROADMAP FOR ORGANIZATION DEVELOPMENT Focused on dwarfism. With 10,000+ families in America we can raise $10M+/year. Our immediate goal is to raise $200K/year. Growing Stronger is the first dedicated non-profit supporting research on dwarfismExamples of existing non-profits contributing to science Focused on healthy babies. Raised $187M in 2010. Spent $28M on research to prevent premature birth. Funded some Achondroplasia projects. Raised $297M in 2010. Invested $72M in research which includes 103 new grants to researchers in academic institutions. Supported 347 research projects in the US, Canada and 9 other countries Focused on breast cancer. Raised $400M in 2010. Invested $75M in research
  12. 12. RESEARCH STRATEGIES FGFR3 Bone FGFR3 Mutant Abnormal Growth Mutant Expression Protein Signals Machinery Gene (suppresses bone growth) impaired 2. Disable the 3. Block/Reduce1. Block expression of Mutant effects of the mutant gene Protein mutant protein 4. Modify the Growth Machinery to account for We currently are supporting four FGFR3 defect different strategies in parallel
  13. 13. Strategy 1 Block expression of mutant gene This is a potentially exciting option. Targets have one normal copy of FGFR3 that can be rescued. Recent developments in siRNA make this an attractive solution for silencing the mutant copy. siRNA is a popular strategy and has worked in many conditions
  14. 14. Strategy 2a Disable Mutant Protein Very promising strategy used in Cancer treatment. Add inhibitor that reduces the protective effect of chaperone protein on mutant FGFR3 Strategy works on Achondroplasia cells Currently being tested in mice Currently funded. Will get more idea about potential success next year
  15. 15. Strategy 2b Disable Mutant Protein Small molecule inhibitor of FGFR3 Analog of NF449 Strategy works on improving growth in cells This approach has been used in other conditions and it is approved Very new approach. Needs more basic research Potential research area for support
  16. 16. STRATEGY 3A Block/Reduce effects of the mutant protein New pathway identified that may be important to bone growth effect of FGFR3 signals In April 2010 discovered new processing step in FGFR3 protein It is possible to leverage existing drugs developed to treat other diseases for the treatment of Achondroplasia (FGFR3 protein)
  17. 17. STRATEGY 3B Block/Reduce effects of the mutant protein Research done in Japan. Strategy looks promising Adding a protein (CNP) has shown progress in mice Drug companies focused on using CNP BioMarin - SF based. Very close to clinical trials Chugai Pharmaceuticals - Japanese firm. No update. Funded by BioMarin and Chugai Clinical Trials Announced Jan 2012
  18. 18. STRATEGY 4 Modify the Growth Machinery to account for FGFR3 defect Need to understand basic mechanisms for how FGFR3 affects bone growth This basic research will help understand what exactly FGFR3 is doing to cartilage cells. With more understanding we will be able to explore other pathways and discover new potential therapies
  19. 19. UNDER DISCUSSION / EARLY STUDIES Case Western University Spain FranceSummary:Achondroplasia is an active area of research with severalsimultaneous efforts. We are on the edge of a breakthrough.We need to accelerate lab testing to maintain momentumand reach the end goal – an approved therapy.
  20. 20. NEXT STEPS Raise $600K to enable lab testing Based on years of research multiple strategies are ready to enter lab testing. If we push we can get into drug development, clinical trials and approval within next 2-6 years. Directly impacts kids under 16 Use of Funds Support mentored scientists in 2 labs for the next 3 years Will spend $200K/ year for the next 3 years $100K pays for a mentored scientist and supplies, maintenance of mouse models of achondroplasia for one year Current status: Raised $156K/year. Need to raise $200K/year by Feb 2012
  21. 21. Non-profit operated viaFUNDRAISING Plan Approach friends and family donors then expand Logistics Donate to non-profit 501c3 Funds will be delivered to the target labs on a bi-annual basis Follow-up We will send a bi-annual report for the next 3 years providing you with up-to-date status on supporting current research
  22. 22. DONOR SHEETDonate via Check or Wire Payee: Growing Stronger Research Fund # 1038759 Mail to: Fidelity® Charitable Gift Fund Please remember to include your name and return P.O. Box 770001 address to receive a tax deduction receipt Cincinnati, OH 45277-0053 If you prefer to wire funds please use the following info: JPMorgan Chase Bank, New York, NY, ABA Number: 021000021 For credit to: National Financial Services LLC. Account: 066196-221 For benefit of: Fidelity® Charitable Gift Fund, Z– 97– 000442. For further credit to: Growing Stronger Research Fund # 1038759
  23. 23. CONTACT For more information and how to donate please contact: Amer Haider, Munira Shamim Invested parents email:, cell: 408-234-0520, 408-828-3387