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-418 – Glycosylation substrate for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9)
BBP-631 – AAV5 gene therapy for congenital adrenal hyperplasia (CAH)
BBP-812 – AAV9 gene therapy for Canavan disease