NCATS | Technology Transfer

2-substituted Pyridines and Their Methods for Inhibiting BMP Signaling for the Treatment of Fibrodysplasia Ossificans Progressiva

Read more about 2-substituted Pyridines and Their Methods for Inhibiting BMP Signaling for the Treatment of Fibrodysplasia Ossificans Progressiva

This technology includes the use of a new class of molecules (nanomolar ALK2 inhibitor) to impede bone morphogenetic proteins (BMP) signaling for the treatment of Fibrodysplasia ossificans progressiva (FOP). FOP is a rare disease, characterized by malformation of the great (big) toes during embryonic development. Individuals with FOP have an identical heterozygous activating mutation (R206H) in the gene encoding ACRV1 (also known as ALK2), a BMP type 1 receptor.

Naphthalene-containing Selective Inhibitors of BMP type 1 Receptors for the Treatment of Fibrodysplasia Ossificans Progressiva

Read more about Naphthalene-containing Selective Inhibitors of BMP type 1 Receptors for the Treatment of Fibrodysplasia Ossificans Progressiva

This technology includes the use of a new class of molecules (nanomolar ALK2 inhibitor) to impede bone morphogenetic proteins (BMP) signaling for the treatment of Fibrodysplasia ossificans progressiva (FOP). FOP is a rare disease, characterized by malformation of the great (big) toes during embryonic development. Individuals with FOP have identical heterozygous activating mutation (R206H) in the gene encoding ACRV1 (also known as ALK2), a BMP type 1 receptor.

Pyrazolo[1,5-a]pyrimidine Derivatives as Selective ALK Kinases Inhibitors for Inhibition of the Bone Morphogenetic Proteins Signaling Pathway for Treatment of Fibrodysplasia Ossificans Progressiva

Read more about Pyrazolo[1,5-a]pyrimidine Derivatives as Selective ALK Kinases Inhibitors for Inhibition of the Bone Morphogenetic Proteins Signaling Pathway for Treatment of Fibrodysplasia Ossificans Progressiva

This technology includes compounds which are selective inhibitors of anaplastic lymphoma kinases (ALK1, ALK2, ALK3 and ALK6), which inhibit these ALKs with low nanomolar potency. These compounds could be developed as a treatment of Fibrodysplasia ossificans progressiva (FOP) and other BMP-related diseases. FOP is a rare congenital disease with no current treatment options. Since the disease is driven by constitutively active ALK2, inhibition of ALK2 would be like hitting the Achilles’ heel of the disease and would potentially be an efficacious therapy for FOP patients.