“Cure Entrepreneurs”<br />T. Craig Benson<br />Accelerating a Cure Through Prevention: <br />A Carrier Screening Strategy<...
 One Girl …<br />
 Juvenile Batten Disease<br />
 Moving Forward … Taking Action<br />
 Beyond Batten Disease Foundation<br />MISSION: <br />To eradicate Batten disease. <br />
 Mission: Two Pronged Approach<br />Research and Development: Raise awareness and money to accelerate research to find a c...
 Research and Development<br />$2.5 Million grant to Neurological Research Institute at Texas Children’s Hospital, Houston...
 Why are they orphans?<br />
 Some Statistics<br />Batten Disease: 750<br />Adrenoleukodystrophy: 13,500<br />Hurler Syndrome: 500<br />Canavan Disease...
 How it’s inherited<br />
Autosomal Recessive Disorders…<br />You have heard of:<br />Cystic Fibrosis<br />Sickle Cell Anemia<br />Tay-Sachs<br />
Autosomal Recessive Disorders…<br />You probably have not heard of:<br /><ul><li>Batten
Gaucher
Alpha-Mannosidosis
Krabbe
Usher Syndrome</li></li></ul><li> Current State of Carrier Screening<br />Tests for 450+ individual devastating diseases e...
Not readily available
Unfamiliar to medical professionals
Not reimbursed as a screening test</li></li></ul><li> Ideally, the test would be:	<br />Inexpensive<br />All-inclusive (45...
 Carrier Screening<br />The eradication of Tay-Sachs disease from the<br />North American Ashkenazi Jewish community.<br /...
The Success Story of Carrier Screening Tests<br />  “Tay-Sachs is the longest running, population-based program designed t...
The Success Story of Carrier Screening Tests<br />  “The Tay-Sachs Disease experience can be viewed as a prototypic effort...
 BBDF and NCGR Present<br />Universal Carrier Screening Test<br />: Under $500<br />450+ Diseases<br />
 Carrier Screening Impact<br />Cases Prevented<br />675<br />12,150<br />450<br />38,250<br />40,500<br />14,400  <br />11...
 Project Timeline<br />March 2010<br />June 20, 2008<br />Phase III Completed<br />March 28, 2008<br />November 15, 2008<b...
 Moore’s Law Turbocharged<br />$100,000<br />$1,000<br />Cost of Catastrophic Gene Analysis over time<br />$25K<br />$3K<b...
Test Process<br />
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  • Here's a great resource for that Hillary. May all your dreams come true!

    http://www.jacobscure.org
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  • Canavan disease is an awful disease. I hope they find a cure for these kids soon!
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  • Batten oss 727 -bw changes

    1. 1. “Cure Entrepreneurs”<br />T. Craig Benson<br />Accelerating a Cure Through Prevention: <br />A Carrier Screening Strategy<br />President and <br />Chief Executive Officer<br />Founder<br />
    2. 2. One Girl …<br />
    3. 3. Juvenile Batten Disease<br />
    4. 4. Moving Forward … Taking Action<br />
    5. 5. Beyond Batten Disease Foundation<br />MISSION: <br />To eradicate Batten disease. <br />
    6. 6. Mission: Two Pronged Approach<br />Research and Development: Raise awareness and money to accelerate research to find a cure for Batten disease. <br />2. Prevention: Develop an easy and inexpensive blood test to detect the gene mutations for Batten disease, and hundreds of other rare conditions like it, that claim the lives of thousands of children each year. <br />
    7. 7. Research and Development<br />$2.5 Million grant to Neurological Research Institute at Texas Children’s Hospital, Houston, TX <br />Danielle Kerkovich, Ph.D.<br />
    8. 8. Why are they orphans?<br />
    9. 9. Some Statistics<br />Batten Disease: 750<br />Adrenoleukodystrophy: 13,500<br />Hurler Syndrome: 500<br />Canavan Disease: 42,500<br />Spinal Muscular Atrophy: 45,000<br />Usher Syndrome: 16,000<br />Charcot Marie Tooth Disease: 125,000<br />Pompe Disease:  6,800<br /> There are approximately 4,250 rare genetic diseases1 that affect 3 to 4% of all children.2 <br />Online Mendelian Inheritance in Man <br />European Organisation for Rare Diseases<br />
    10. 10. How it’s inherited<br />
    11. 11. Autosomal Recessive Disorders…<br />You have heard of:<br />Cystic Fibrosis<br />Sickle Cell Anemia<br />Tay-Sachs<br />
    12. 12. Autosomal Recessive Disorders…<br />You probably have not heard of:<br /><ul><li>Batten
    13. 13. Gaucher
    14. 14. Alpha-Mannosidosis
    15. 15. Krabbe
    16. 16. Usher Syndrome</li></li></ul><li> Current State of Carrier Screening<br />Tests for 450+ individual devastating diseases exist and are definitive, but they are:<br /><ul><li>Prohibitively expensive ($500 - $1,300 per person / per disease)
    17. 17. Not readily available
    18. 18. Unfamiliar to medical professionals
    19. 19. Not reimbursed as a screening test</li></li></ul><li> Ideally, the test would be: <br />Inexpensive<br />All-inclusive (450+ devastating diseases)<br />Administered to prospective parents<br />Widely available<br />
    20. 20. Carrier Screening<br />The eradication of Tay-Sachs disease from the<br />North American Ashkenazi Jewish community.<br />First community-based screening program<br />DorYeshorim Founded<br />Near total disappearance of Tay-Sachs disease from the Jewish community<br />1971<br />1999<br />1983<br />
    21. 21. The Success Story of Carrier Screening Tests<br /> “Tay-Sachs is the longest running, population-based program designed to prevent a lethal genetic disease. Screening has reduced the number of Tay-Sachs cases in the U.S. and Canada by 90%.”<br />- Genome News Network <br />
    22. 22. The Success Story of Carrier Screening Tests<br /> “The Tay-Sachs Disease experience can be viewed as a prototypic effort for public education, carrier testing, and reproductive counseling for avoiding fatal childhood disease.”<br />- Genetic testing 1998;2(4):271-92 <br />
    23. 23. BBDF and NCGR Present<br />Universal Carrier Screening Test<br />: Under $500<br />450+ Diseases<br />
    24. 24. Carrier Screening Impact<br />Cases Prevented<br />675<br />12,150<br />450<br />38,250<br />40,500<br />14,400 <br />112,500<br />6,120<br />Batten Disease:<br />Adrenoleukodystrophy:<br />Hurler Syndrome:<br />Canavan Disease:<br />Spinal Muscular Atrophy:<br />Usher Syndrome:<br />Charcot Marie Tooth Disease:<br />Pompe Disease:<br />750<br /> 13,500<br /> 500<br /> 42,500<br /> 45,000<br /> 16,000 <br /> 125,000<br /> 6,800<br />225,000<br />Total: <br />
    25. 25. Project Timeline<br />March 2010<br />June 20, 2008<br />Phase III Completed<br />March 28, 2008<br />November 15, 2008<br />Christiane Diagnosis<br />BBDF/NCGR Phase I <br />Project Initiated<br />Final site selection<br />November 1, 2010<br />May 2011<br />BBDF Formed<br />Phase II Completed<br />Product launch <br />August 22, 2008<br />February 28, 2009<br />
    26. 26. Moore’s Law Turbocharged<br />$100,000<br />$1,000<br />Cost of Catastrophic Gene Analysis over time<br />$25K<br />$3K<br />$500<br />?<br />2005<br />2007<br />2009<br />2010<br />2011<br />
    27. 27. Test Process<br />
    28. 28. One of the largest gene analysis centers in the world that will:<br /><ul><li>Screen carriers for 450+ inherited diseases of children
    29. 29. Become a Human Genetics branch of the National Center for Genome Resources</li></ul> What we’ll deliver<br /><ul><li>Apply sequencing technology to other orphan diseases: create population database
    30. 30. Bring these benefits to the entire world</li></li></ul><li> Sustainability<br />450+ Diseases: Under $500<br />A percentage of the test proceeds will be retained to permanently fund research for treatments and cures.<br />
    31. 31. Sustainability <br />
    32. 32. Be a “Cure Entrepreneur”<br /><ul><li> Find and BECOME a leading expert
    33. 33. Understand your assets and network
    34. 34. Leverage the above
    35. 35. Join and lead efforts with other constituents
    36. 36. Identify opportunities, obstacles and set clear targets
    37. 37. SUSTAINABILITY is key … it’s a long road</li></li></ul><li>“Cure Entrepreneurs”<br />T. Craig Bensoncbenson@beyondbatten.org<br />A Daughter's Diagnosis:<br />((TBD))<br />President and <br />Chief Executive Officer<br />Founder<br />
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