Human Genetics For Target Discovery

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JTMulligan

Technologie Business

Genetic Discovery of Drug Targets ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

Example 1: A Target for Osteoporosis ,[object Object],[object Object],[object Object],[object Object],Normal Sclerosteosis

Genetic Mapping to 17q21 Shared Disease Haplotype in black Genetic mapping of 22 families defined a region of chromosome 17q21

Candidate Gene Identification by Sequencing (Circa 1997) ,[object Object],[object Object],[object Object]

Functional Confirmation of Mutation ,[object Object],[object Object],[object Object],[object Object]

“ Sclerostin” is a Cystine Knot Protein ,[object Object],[object Object],[object Object],[object Object],Noggin protein (family member)

Patent on Antibodies That Block Sclerostin ,[object Object],[object Object]

Anti-Sclerostin Antibody in Clinical Trials

IL23 Pathway as a Drug Development Target ,[object Object],[object Object],[object Object],[object Object]

IL-23 Antagonist in Clinical Trials This work pre-dated the genetic mapping results

“ Drug Surrogate” Mutations ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

Mapping Strategy ,[object Object],[object Object],[object Object],[object Object],[object Object]

Examples ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

Changing the Economics of Drug Development ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

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