12/19/2025
As 2025 comes to a close, we’re reflecting on a year defined by scientific progress and the strength of the community that makes it possible.
This year, we advanced our Phase 2 clinical trial of RCT2100, including FDA clearance to study the therapy in combination with ivacaftor, an important step toward expanding options for people with cystic fibrosis who currently have no benefit from approved modulators.
We strengthened our foundation with more than $29M in new financing, expanded support from the , and a new research collaboration with to explore novel LNP delivery for antisense oligonucleotides (ASOs).
But the real story of 2025 was the people — the investigators, advocates, families, and colleagues whose partnership and resilience guided every milestone.
As we head into 2026, we remain grateful for the trust of the communities we serve and energized by the shared belief that our innovative science, pursued with care and intention, can expand the future of genetic medicine.
