Target Validation and Lead Discovery: From a Biological Claim to a Molecule · seed 1 · A4, ink-friendly. The answer key prints on its own page for grown-ups.

From disease gene to a molecule that works

Science · Biochemistry & Molecular Biology · ages 23-24
Name ______________________   Date ____________

  1. What does genetic evidence for a target leave open?

    • Whether the target matters in disease
    • Whether a drug can reach and modulate it safely
    • Whether the gene exists

  2. What question does target validation answer?

    • Whether changing the target changes disease and a molecule can reach it
    • Which company should develop the drug
    • What the drug will be named

  3. Agonists push a receptor while antagonists block it.

    Circle one:   True   False

  4. Chemical validation shows clean modulation in cells. What stays open?

    • Whether patients benefit from it
    • Whether the molecule binds anything
    • Whether modulation is possible

  5. A hit looks great in one well. What is the next honest step?

    • Rename it and publish
    • Call it a lead and scale up chemistry
    • Confirm it in fresh assays with dose response and counterscreens

  6. A candidate is potent and selective but never reaches free levels in tissue. Your verdict?

    • Advance it as a medicine
    • Treat it as a reagent, not a medicine
    • Fix it by raising the dose without limit

  7. High affinity alone guarantees a safe, effective drug.

    Circle one:   True   False

  8. A program has only genetic evidence and wants full chemistry spending now. What do you advise?

    • Skip validation and screen broadly
    • Spend now; genetics is enough
    • Get chemical validation first: reach and safe modulation are unproven
LightMySky · lightmysky.comW1-mt_xHWq5Rg85r-s1

Answer key

For grown-ups. Fold this page away before handing over the rest.

From disease gene to a molecule that works W1-mt_xHWq5Rg85r-s1

  1. Whether a drug can reach and modulate it safely · Gene links do not guarantee a reachable, safe drug target.
  2. Whether changing the target changes disease and a molecule can reach it · Validation covers the disease link and the reachability of the target.
  3. True · The dose response curve shows push versus block directly.
  4. Whether patients benefit from it · Possible modulation is one leg; patient benefit is the other.
  5. Confirm it in fresh assays with dose response and counterscreens · Single-well promise is mostly artifacts until the gates clear it.
  6. Treat it as a reagent, not a medicine · Without exposure at the target, potency cannot act in a body.
  7. False · Affinity is one seat-winning contest; selectivity and exposure decide the rest.
  8. Get chemical validation first: reach and safe modulation are unproven · Serious spending waits until both validation legs stand.
Worksheet · LightMySky