Operating on DNA is more like surgery than medicine

• Baby KJ: custom gene edit for CPS1 deficiency, 6 months, <$1M, success.
  • New paradigm: personalized genetic intervention, not a drug.
• Genetic surgery ≠ gene therapy — targets one patient’s exact mutation.
  • Tools: CRISPR-Cas9, base editors, prime editors, engineered delivery.
• FDA treats every novel mutation edit as a new drug: $100M+ and a decade.
  • 10,000 rare genetic diseases, 30M Americans affected, 95% lack approved treatments.
• FDA proposed “plausible mechanism” pathway (late 2025) for ultra-rare cases.
  • Also proposed “platform technology designation” for reusable components.
• Conde proposes SAGE: certify centers, not individual edits — modeled on CLIA.
  • Centers demonstrate multidisciplinary review, manufacturing, outcomes tracking.
  • Patient pipelines replace product pipelines; no commercial-scale trial required.
• SAGE centers anchor in academic hubs (Boston, Philly, SF); can’t offshore.
  • Rare-disease “genetic data exhaust” feeds broader therapeutic programs.
• Cost ~$1M per case — like open-heart surgery; outcomes-based reimbursement emerging.

Jorge Conde, General Partner — a16z Bio + Health (therapeutics, diagnostics, life sciences tools) · 2026-01-28 · Read on a16z.com