Acoramidis (AG10) – TTR stabilizer for transthyretin amyloidosis (ATTR)
Patient and family experience with transthyretin amyloid cardiomyopathy (ATTR-CM) and polyneuropathy (ATTR-PN) amyloidosis: results of two focus groups
Rintell, D. et al. Orphanet J Rare Dis. 2021. 16:70. https://doi.org/10.1186/s13023-021-01706-7.
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 pro-drug for limb-girdle muscular dystrophy 2I/R9 (LGMD2I/R9)
KRAS mutant-driven cancers
BBP-398 – SHP2 inhibitor for tumors driven by RAS and receptor tyrosine kinase mutations
BBP-671 – PanK activator for pantothenate kinase-associated neurodegeneration (PKAN) and organic acidemias (OA)
Pantothenate kinase activation relieves coenzyme A sequestration and improves mitochondrial function in mice with propionic acidemia
Subramanian C, et al. Sci Transl Med. 2021 Sep 15;13(611):eabf5965. doi: 10.1126/scitranslmed.abf5965.
A therapeutic approach to pantothenate kinase associated neurodegeneration
Sharma LK, et al. Nat Commun. 2018 Oct 23;9(1):4399. doi: 10.1038/s41467-018-06703-2. PMID: 30352999; PMCID: PMC6199309.
BBP-631 – AAV5 gene therapy for congenital adrenal hyperplasia (CAH)
BBP-812 – AAV9 gene therapy for Canavan disease
NULIBRY™ (fosdenopterin) – Synthetic cPMP for MoCD Type A
BridgeBio
presentations & publications
BridgeBio is committed to letting science speak. Here we have gathered a selection of publications relevant to our programs.
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