Sharp Edge Labs: Discovering Drugs to Treat Genetic Disorders of Protein Trafficking.

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Off the Shelf
  • Monogenic Diseases
    Caused by a single gene alteration, monogenic diseases represent an area of high unmet need, and also of great therapeutic opportunity.
  • Trafficking Defects
    Many monogenic diseases result from a mutation that causes improper trafficking, the failure of the mutated protein to get to the right place inside the cell.
  • Patient-Driven Discovery
    Stem-Cell technology allows patients to participate in drug discovery programs at their earliest stages.
On the Bench
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