The Rothberg Institute

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The Rothberg Institute

• Drugs For Childhood Diseases
• www.childhooddiseases.org
• Non Profit Organization

John Chant 10/10/03
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TRI Organization
Clinical
Preclinical Small Molecules Antibodies (Animal
Studies)
AML LAM SEGA
Angiofibroma
Cell Line Immortalization AML, LAM, SEGA
Target Identification Microarrays Pathway
Elucidation Serum profiling, IHC
CommunityTSC Target and Library Prep Virtual
Library Screening Data Analysis Community Forums
Screening Cell Based Screens
Therapeutic Antibodies Fully Human Monoclonal
Antibodies
Chemical Libraries 10,000 drug-like 5000 natural
products 2000 known active drugs
John Chant 10/10/03
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TRI Target Focus

• Rheb Farnesyl Transferase Inhibitors (Wyeth
  Merck)
• m-Tor (FRAP) Rapamycin derivatives (Wyeth
  Ariad)
• S6 Kinase Inhibitors unknown (Amphora
  others???)
• VEGF Avastin (Antibody) (Genentech)
• VEGF-Receptor Compounds in clinical trials (???)
• Raf Onyx/Bayer compounds (Bayer)
• PDGF-D CR002 Antibody (CuraGen)
• NMB CR011 Antibody (CuraGen)
• Other small molecule inhibitors
• EGFR Iressa (AstraZeneca)
• Ab and other kinases Gleevec (Novartis)

John Chant 10/10/03
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TRI 2004 Goals

• Research
• Survey tissue and sera for therapeutic targets
  and markers
• Lead for new drug that specifically kills AML,
  LAM, or SEGAs
• Fully Human Monoclonal against LAM or AML target
• Preclinical 4 New Animal Studies
• Anti-VEGF Ab and Small molecule
• FTase Inhibitors
• CR002
• Clinical
• Catalyze 1 New Clinical study
• Build comprehensive tissue and serum bank
• Courage Fund
• Sponsor 4 Animal Studies

John Chant 10/10/03
5
TRI 2005 Goals

• Preclinical
• 4 Additional Animal Studies
• Clinical
• Initiate one new clinical trial in collaboration
  with one or more TSC clinical site(s)
• Transition for Phase IIb/III Study

John Chant 10/10/03