This study evaluates the efficacy of BLU-782, a selective inhibitor of activin receptor-like kinase 2 (ALK2), in preventing heterotopic ossification (HO) in a mouse model of fibrodysplasia ossificans progressiva (FOP). The research aims to determine whether BLU-782 can inhibit aberrant bone formation associated with FOP.
Prophylactic oral dosing of BLU-782 significantly reduced edema and prevented cartilage and heterotopic bone formation in both muscle and bone injury models in the ACVR1 R206H mouse. These findings suggest that BLU-782 may be a promising candidate for preventing HO in FOP patients.
The study utilized a conditional Knockin mouse model developed in collaboration with genOway. This model harbors the human ACVR1 R206H mutation, which is the most common variant causing FOP. The design includes a loxP-flanked STOP cassette upstream of the mutant allele, allowing for tissue-specific expression of the mutation upon Cre recombinase-mediated recombination. This configuration enables precise control over the temporal and spatial activation of the mutant ACVR1 gene, facilitating the study of its role in HO development.
Fibrodysplasia ossificans progressiva, Heterotopic ossification, Bone morphogenetic protein pathway, Genetic disorders, Musculoskeletal diseases
Conditional Knockin, LoxP-flanked STOP cassette, Cre recombinase-mediated recombination, Tissue-specific expression, Humanized mutation model
From model design to experimental results
Tailor-made solutions adapted to scientific questions
Comprehensive dataset package
Generated with biopharma partners and in-house
Scientific follow-up and advice along the project
Collaborative approach for problem solving and development of innovative models
Breeding facilities in US and Europe
Certified health status from professional breeders
