Last Week in Biomedicine | Jun 29–Jul 3 | Part 1 of 2: CRISPR for Toddlers, KRAS Lung Cancer Win

CRISPR gene editing crossed a major line this week: the FDA now allows Casgevy — the first CRISPR therapy of any kind — for children as young as 2 with sickle cell disease, meaning we can potentially prevent a lifetime of pain crises and organ damage instead of treating them after the fact. Part 1 also digs into a rare kind of trial where a new lung cancer drug takes on the already-approved competition, and a New England Journal of Medicine paper on the first oral pill for a form of childhood dwarfism.

In Part 1 we cover:

1. Vertex Pharmaceuticals / CRISPR Therapeutics — Casgevy (exagamglogene autotemcel) for sickle cell disease and transfusion-dependent beta thalassemia in children ages 2 and up (FDA approval — label expansion)

2. Roche / Genentech — divarasib for KRAS G12C-mutant non-small cell lung cancer (Phase 3)

3. BridgeBio Pharma / Kyowa Kirin — oral infigratinib for achondroplasia in children (Phase 3, published in NEJM)

What could this mean for patients? Casgevy in toddlers is the story with the biggest potential ripple effect. Sickle cell disease starts hurting kids in infancy, so treating a 2-year-old before repeated strokes and organ damage sets in could redefine what growing up with sickle cell looks like. It's also a proof point that CRISPR-based cures can be delivered safely in very young children — a green light for other pediatric gene-editing programs in development.

Part 2 drops Thursday morning. Subscribe at TrialBreaks.com.

#biomedicine #clinicaltrials #science #CRISPR #genetherapy #sicklecell #lungcancer #KRAS #achondroplasia #oncology #rarediseases #pediatrics #hematology #NEJM #immunotherapy