Venthera, Inc., a BridgeBio company
BridgeBio is a biopharmaceutical company dedicated to discovering, creating, testing, and delivering transformative medicines for patients with genetic diseases. They focus on scientific innovation, collaboration with academic and industry partners, and a decentralized operating model to develop therapies rapidly and at scale. Their mission is to help patients with genetic conditions by bridging scientific advancements to clinical solutions.
Industries
Nr. of Employees
large (251-1000)
Venthera, Inc., a BridgeBio company
Products
TTR stabilizer small-molecule for transthyretin-mediated amyloid cardiomyopathy
Orally administered small-molecule therapy indicated to reduce mortality and cardiovascular hospitalization in transthyretin-mediated amyloid cardiomyopathy; includes commercial product labeling considerations and documented gastrointestinal adverse reactions.
Low-dose FGFR inhibitor for skeletal dysplasia (achondroplasia, hypochondroplasia)
Oral FGFR3 inhibitor being developed to reduce overactive FGFR3 signaling in children with skeletal dysplasias; advanced into registrational Phase 3 for achondroplasia.
Small-molecule substrate therapy for FKRP-related limb-girdle muscular dystrophy
Oral small-molecule candidate intended to increase glycosylation of alpha-dystroglycan via substrate supplementation to restore or stabilize muscle function in FKRP-mutant patients.
Allosteric calcium-sensing receptor modulator for hypocalcemia and hypoparathyroidism
Small-molecule negative allosteric modulator of the calcium-sensing receptor in global Phase 3 development for autosomal dominant hypocalcemia type 1 and being evaluated in hypoparathyroidism.
AAV gene therapy program for Canavan disease
AAV-based gene replacement program aiming to deliver functional ASPA gene copies to correct metabolic defect in Canavan disease; in clinical Phase 2.
TTR stabilizer small-molecule for transthyretin-mediated amyloid cardiomyopathy
Orally administered small-molecule therapy indicated to reduce mortality and cardiovascular hospitalization in transthyretin-mediated amyloid cardiomyopathy; includes commercial product labeling considerations and documented gastrointestinal adverse reactions.
Low-dose FGFR inhibitor for skeletal dysplasia (achondroplasia, hypochondroplasia)
Oral FGFR3 inhibitor being developed to reduce overactive FGFR3 signaling in children with skeletal dysplasias; advanced into registrational Phase 3 for achondroplasia.
Small-molecule substrate therapy for FKRP-related limb-girdle muscular dystrophy
Oral small-molecule candidate intended to increase glycosylation of alpha-dystroglycan via substrate supplementation to restore or stabilize muscle function in FKRP-mutant patients.
Allosteric calcium-sensing receptor modulator for hypocalcemia and hypoparathyroidism
Small-molecule negative allosteric modulator of the calcium-sensing receptor in global Phase 3 development for autosomal dominant hypocalcemia type 1 and being evaluated in hypoparathyroidism.
AAV gene therapy program for Canavan disease
AAV-based gene replacement program aiming to deliver functional ASPA gene copies to correct metabolic defect in Canavan disease; in clinical Phase 2.
Services
Engagement and negotiations for academic and industry collaborations, asset licensing, portfolio divestments, and co-development arrangements.
Provision of grants, fellowships, and scientific sponsorships for evidence-based education, conferences, and academic training aligned to therapeutic focus areas.
Medical and regulatory assessment of individual patient requests for pre-approval access to investigational therapies, including IRB and safety reporting requirements.
Ongoing collaboration with patients, families, and advocacy organizations to incorporate lived experience into trial design and communications.
Strategic regulatory planning, trial design aligned with agency feedback, and preparation for submissions and designation requests.
Engagement and negotiations for academic and industry collaborations, asset licensing, portfolio divestments, and co-development arrangements.
Provision of grants, fellowships, and scientific sponsorships for evidence-based education, conferences, and academic training aligned to therapeutic focus areas.
Medical and regulatory assessment of individual patient requests for pre-approval access to investigational therapies, including IRB and safety reporting requirements.
Ongoing collaboration with patients, families, and advocacy organizations to incorporate lived experience into trial design and communications.
Strategic regulatory planning, trial design aligned with agency feedback, and preparation for submissions and designation requests.
Expertise Areas
- Clinical trial management
- Gene therapy development
- Small-molecule therapeutics
- Rare disease drug development
Key Technologies
- AAV-based gene therapy
- Small-molecule drug discovery
- Topical and oral formulation development
- Allosteric modulation of receptors