Acoramidis (AG10) – TTR stabilizer for transthyretin amyloidosis (ATTR)
Low-dose infigratinib – FGFR1-3 inhibitor for achondroplasia, hypochondroplasia & other skeletal dysplasias
Encaleret – Ca sensing receptor antagonist for autosomal dominant hypocalcemia type 1 (ADH1)
BBP-671 – PanK activator for pantothenate kinase-associated neurodegeneration (PKAN) and organic acidemias
Statistical genetics and computational genomics
BBP-589 – Recombinant collagen for recessive dystrophic epidermolysis bullosa (RDEB)
BBP-681 – Topical PI3Ka inhibitor for venous malformations (VMs), lymphatic malformations (LMs) and venolymphatic malformations
BBP-711 – GO inhibitor for primary hyperoxaluria type 1 (PH1) and recurrent kidney stone formers