> At a Glance
> – Jennifer Doudna co-founds Aurora Therapeutics to scale bespoke CRISPR fixes like the one that saved baby KJ.
> – New FDA “plausible mechanism pathway” needs data from only a handful of patients, not thousands.
> – Aurora will first target phenylketonuria (PKU), a metabolic disorder affecting 13,500 Americans.
> – Why it matters: Faster approvals mean ultra-rare disease patients could get life-saving edits in months, not years.
A single, custom CRISPR therapy rescued baby KJ from a deadly ammonia buildup last year. Now Aurora Therapeutics, launched by Nobel laureate Jennifer Doudna, wants to repeat that success for thousands more.
From One-Off Cure to Platform
Last February, doctors designed a gene edit for KJ in six months. He went home in June. Aurora will swap the same base-editing components to match 1,000-plus PKU mutations without starting every therapy from scratch.
CEO Edward Kaye, a pediatric neurologist, says the company’s standardized process will let it “leave no mutation behind.”
How the FDA Shortcut Works
- Traditional trials: Hundreds or thousands of patients required
- New pathway: Show consecutive success in a few patients with related bespoke therapies
- Marketing authorization: Granted for the platform, not just one edit
Target Disease: PKU at a Glance
| Statistic | Number |
|---|---|
| US patients | 13,500 |
| Known mutations | 1,000+ |
| Toxic metabolite | Phenylalanine |
Without treatment, PKU stunts brain development and forces a near-zero-protein diet.
Genome scientist Fyodor Urnov, Aurora co-founder, helped build KJ’s guide RNA and now aims to replicate the feat at scale.
Key Takeaways
- Aurora uses base editing, a more precise CRISPR variant
- Each therapy keeps the same delivery system; only the guide RNA changes
- FDA’s new route slashes development time and cost for ultra-rare diseases
If the model works, bespoke gene edits could become the default for fatal single-gene disorders rather than last-ditch miracles.
