25/03/2026
Ottawa, Ontario — Wednesday March 25, 2026
Babies born with rare genetic surfactant disorders face life-threatening breathing problems from their first breath.
Surfactant is the slippery substance that keeps the lungs’ air sacs open, and without it, the lungs cannot function. For infants with surfactant protein C (SP-C) deficiency, treatments are limited and many ultimately require a lung transplant.
A team led by Bernard Thébaud is working to change that. In a new study published in Molecular Therapy, researchers tested a dual action gene therapy designed for one of the most challenging forms of the disease: cases where a harmful, toxic version of the SPC protein is produced alongside the normal version.
“What we see in this model is encouraging and justifies taking the next steps toward clinical trials. Our goal is to translate these findings into a therapy that could meaningfully change the trajectory of SP‑C deficiency for newborns.” – Bernard Thébaud
Using a virus to deliver genetic instructions directly to the lungs, the therapy does two things: it replaces the missing healthy protein and silences the toxic form. A single dose improved lung structure and lung function in newborn mice, reducing the air sac damage that normally worsens over time.
While more research is needed before clinical trials can begin, the findings offer proof of concept that a single treatment could one day modify the course of this disease – giving families hope for a future treatment option.