Baby KJ
FDA Unveils ‘Plausible Mechanism Pathway’ to Accelerate Personalized Therapies, Inspired by Baby KJ
FDA; personalized therapies; gene editing; plausible mechanism pathway; Baby KJ; rare diseases; CRISPR; regulatory innovation
Researchers Publish Roadmap of FDA Interactions After Baby KJ’s Groundbreaking Gene Editing Therapy, as FDA Signals New Pathway for Patient-Specific Treatments
Baby KJ; CRISPR gene editing; CPS1 deficiency; FDA approval pathway; personalized medicine; rare disease; regulatory roadmap; platform trials; N-of-1 therapies; rare disease gene therapies