Pipelines, approvals and first-in-child therapies, from small molecules to gene editing.
Many of the conditions that one-time genetic treatments target first appear in infancy. That puts children at the centre of the field’s scientific, commercial and ethical debates.
Treatments
The first CAR-T therapy approved anywhere was for children and young adults with leukaemia. That history still shapes how the field thinks about paediatric patients.
Treatments
In less than four years, a disease with no treatment gained three, each using a different technology. SMA has become a case study in how modality shapes access.
Treatments
Drugs that correct the underlying protein defect have changed cystic fibrosis. The next question is how early in life they should start.
Treatments
The first approved CRISPR-based medicine treats sickle cell disease from age 12. Extending it to younger children raises questions about conditioning, fertility and timing.
Treatments
Doctors have begun designing genetic medicines for one patient at a time. The science is moving faster than the regulatory and funding models built around it.
