Gene editing unlocks 'niche' rare drugs

CRISPR‑era editing cuts development time and cost enough that treatments for very small patient populations — previously 'too small' to commercialize — become viable. That shifts pharma economics, testing pathways, and who gets prioritized for scarce regulatory attention. — If genome editing makes rare‑disease drugs commercially viable, policymakers and payers will face new questions about pricing, equitable access, and accelerated approval norms.