The Cystic Fibrosis Foundation has awarded a milestone payment to Copernicus Therapeutics, Inc. This milestone payment is part of a research, development and commercialization agreement between Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), a non-profit affiliate of the CF Foundation, and Copernicus to develop gene therapy to treat cystic fibrosis.
Copernicus earned the milestone payment by demonstrating a significant improvement in the level of CFTR gene activity in an animal model. This increased improvement in the level and duration of CFTR gene activity represents an important step in the path to developing a clinically relevant therapy.
Copernicus' unique, non-viral nanoparticle formulation is intended to deliver a normal copy of the CF gene to the affected lung cells of CF patients, and may provide a therapy that treats the root cause of CF regardless of the specific mutations resulting in CF. Copernicus' first clinical trial, supported by CFFT, demonstrated the desired safety profile and encouraging biological changes were observed.