KRAS mutant-driven cancers
Acoramidis (AG10) – TTR stabilizer for transthyretin amyloidosis (ATTR)
Encaleret – Ca sensing receptor antagonist for autosomal dominant hypocalcemia type 1 (ADH1)
Low-dose FGFR1-3 inhibitor for achondroplasia – infigratinib
BBP-418 – Glycosylation substrate for limb-girdle muscular dystrophy type 2i (LGMD2i)
BBP-589 – Recombinant collagen for recessive dystrophic epidermolysis bullosa (RDEB)
NULIBRY™ (fosdenopterin) – Synthetic cPMP for MoCD Type A
High-dose infigratinib – FGFR1-3 inhibitor for FGFR+ cancers
BBP-398 – SHP2 inhibitor for tumors driven by RAS and receptor tyrosine kinase mutations
BBP-631 – AAV5 gene therapy for congenital adrenal hyperplasia (CAH)
BBP-812 – AAV9 gene therapy for Canavan disease
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