Eidos Therapeutics
BridgeBio Pharma is a biopharmaceutical company dedicated to discovering, creating, testing, and delivering transformative medicines for patients suffering from genetic diseases. With a focus on genetic disorders and cancers with clear genetic drivers, BridgeBio aims to develop breakthrough therapies at an unprecedented scale by leveraging scientific advancements and a unique operating model that emphasizes speed, scale, and scientific rigor.
Industries
Nr. of Employees
medium (51-250)
Eidos Therapeutics
Products
AAV9-based systemic IV gene therapy candidate for Canavan disease (investigational)
An investigational intravenous AAV9 gene therapy designed to reduce pathological biomarkers and improve neurodevelopmental motor outcomes in pediatric patients with Canavan disease.
cPMP substrate replacement therapy for Molybdenum Cofactor Deficiency Type A (developmental/regulated submission)
A first-in-class cyclic pyranopterin monophosphate substrate replacement therapy intended to restore molybdenum cofactor synthesis and prevent accumulation of neurotoxic metabolites in MoCD Type A.
Oral small-molecule CaSR antagonist for ADH1 (investigational, Phase 3)
An orally administered negative allosteric modulator of the calcium-sensing receptor developed to increase PTH secretion and normalize serum and urinary calcium in patients with ADH1.
AAV9-based systemic IV gene therapy candidate for Canavan disease (investigational)
An investigational intravenous AAV9 gene therapy designed to reduce pathological biomarkers and improve neurodevelopmental motor outcomes in pediatric patients with Canavan disease.
cPMP substrate replacement therapy for Molybdenum Cofactor Deficiency Type A (developmental/regulated submission)
A first-in-class cyclic pyranopterin monophosphate substrate replacement therapy intended to restore molybdenum cofactor synthesis and prevent accumulation of neurotoxic metabolites in MoCD Type A.
Oral small-molecule CaSR antagonist for ADH1 (investigational, Phase 3)
An orally administered negative allosteric modulator of the calcium-sensing receptor developed to increase PTH secretion and normalize serum and urinary calcium in patients with ADH1.
Services
Clinical development and global trial execution
End-to-end clinical program management across Phase 1–3 including operational execution, trial design, and use of external natural history comparators.
Regulatory strategy and submission support
Development and management of global regulatory strategies and preparation of major regulatory submissions (IND/CTA/NDA/MAA) and interactions with health authorities.
Medical affairs, KOL engagement, and scientific communications
KOL mapping, advisory board execution, speaker program management, field MSL campaigns, and scientific exchange to support product development and launch.
HEOR, payer engagement, and market access strategy
Evidence generation, economic modeling, HTA support, and payer-facing value proposition development to enable access and reimbursement.
Genetic testing and counseling program (no-charge eligibility program)
Provision of targeted genetic testing and counseling programs for suspected hereditary hypoparathyroidism and related disorders to enable diagnosis and trial eligibility.
Quality systems, document control, and training services
Development and maintenance of document control processes, EDMS and LMS administration, GxP compliance support, and audit readiness activities.
Clinical development and global trial execution
End-to-end clinical program management across Phase 1–3 including operational execution, trial design, and use of external natural history comparators.
Regulatory strategy and submission support
Development and management of global regulatory strategies and preparation of major regulatory submissions (IND/CTA/NDA/MAA) and interactions with health authorities.
Medical affairs, KOL engagement, and scientific communications
KOL mapping, advisory board execution, speaker program management, field MSL campaigns, and scientific exchange to support product development and launch.
HEOR, payer engagement, and market access strategy
Evidence generation, economic modeling, HTA support, and payer-facing value proposition development to enable access and reimbursement.
Genetic testing and counseling program (no-charge eligibility program)
Provision of targeted genetic testing and counseling programs for suspected hereditary hypoparathyroidism and related disorders to enable diagnosis and trial eligibility.
Quality systems, document control, and training services
Development and maintenance of document control processes, EDMS and LMS administration, GxP compliance support, and audit readiness activities.
Expertise Areas
- Clinical trial management
- Gene therapy development
- Small-molecule drug development
- Regulatory strategy and global submissions
Key Technologies
- AAV-based systemic gene therapy (IV delivery)
- Small-molecule negative allosteric modulation (GPCR antagonists)
- Biomarker assays for N-acetylaspartate (urine/CSF/brain)
- T2-weighted MRI for myelination assessment