Low-dose FGFR1-3 inhibitor for achondroplasia – infigratinib
BBP-589 – Recombinant collagen for recessive dystrophic epidermolysis bullosa (RDEB)
Statistical genetics and computational genomics
BBP-671 – PanK activator for pantothenate kinase-associated neurodegeneration (PKAN) and organic acidemias
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