Technology ID
TAB-3598
Novel ACRV1/ALK2 Inhibitors and Methods for Inhibiting BMP Signaling for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP)
E-Numbers
E-198-2016-0
Lead Inventor
Huang, Wenwei (NCATS)
Co-Inventors
Jiang, Jian-kang (NCATS)
Shamim, Khalida (NCATS)
Sanderson, Philip (NCATS)
Zheng, Wei (NCATS)
Huang, Xiuli (NCATS)
Tawa, Gregory (NCATS)
Alimardanov, Asaf (NCATS)
Lee, Arthur (NCATS)
Huang, Junfeng (NCATS)
Applications
Therapeutics
Research Materials
Therapeutic Areas
Endocrinology
Lead IC
NCATS
ICs
NCATS
This technology includes the identification and use of novel ACRV1/ALK2 inhibitors for the treatment of fibrodysplasia ossificans progressiva (FOP), an autosomal-dominant rare disease that affects one person in every 1-2 million. FOP is characterized by malformation of the great (big) toes during embryonic development and by progressive heterotopic endochondral ossification (HEO) postnatally, which leads to the formation of a second skeleton of heterotopic bone. Individuals with the classical features of FOP have the identical heterozygous activating mutation (R206H) in the gene encoding ACRV1 (also known as ALK2), a BMP type 1 receptor.
Commercial Applications
Further clinical work could establish the novel ACRV1/ALK2 inhibitors for the treatment of fibrodysplasia ossificans progressiva (FOP). Potent ALK2 inhibitors could conceivably be used prophylactic, acute or chronic therapy for patients with FOP as well as disorders associated with altered BMP signaling.
Competitive Advantages
There are no effective treatments for fibrodysplasia ossificans progressiva (FOP).
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