ML Bio Solutions Inc
BridgeBio Pharma is a biopharmaceutical company dedicated to discovering, creating, testing, and delivering transformative medicines for patients suffering from genetic diseases and cancers with clear genetic drivers. Founded in 2015, it leverages scientific advancements to develop breakthrough medicines at an unprecedented scale, focusing on well-characterized genetic diseases and utilizing a unique, entrepreneurial operating model to accelerate drug development and delivery.
Industries
Nr. of Employees
small (1-50)
ML Bio Solutions Inc
3160 Porter Drive, Suite 250, Palo Alto, CA, 94304
Products
Clinical-stage AAV9 intravenous gene therapy candidate for Canavan disease
A systemically administered AAV9 vector gene therapy developed to replace or restore activity of a deficient enzyme in Canavan disease; evaluated for safety, tolerability, biomarker reduction, and developmental outcomes in pediatric patients.
Clinical-stage small-molecule CaSR antagonist for ADH1
An investigational oral small molecule that acts as a negative allosteric modulator of the calcium-sensing receptor to normalize serum and urinary calcium in patients with autosomal dominant hypocalcemia driven by CASR gain-of-function variants.
cPMP substrate-replacement therapy for MoCD Type A (NDA accepted)
A first-in-class cyclic pyranopterin monophosphate (cPMP) substrate-replacement therapy developed to restore molybdenum cofactor synthesis and reduce neurotoxic metabolite accumulation in molybdenum cofactor deficiency Type A.
Clinical-stage AAV9 intravenous gene therapy candidate for Canavan disease
A systemically administered AAV9 vector gene therapy developed to replace or restore activity of a deficient enzyme in Canavan disease; evaluated for safety, tolerability, biomarker reduction, and developmental outcomes in pediatric patients.
Clinical-stage small-molecule CaSR antagonist for ADH1
An investigational oral small molecule that acts as a negative allosteric modulator of the calcium-sensing receptor to normalize serum and urinary calcium in patients with autosomal dominant hypocalcemia driven by CASR gain-of-function variants.
cPMP substrate-replacement therapy for MoCD Type A (NDA accepted)
A first-in-class cyclic pyranopterin monophosphate (cPMP) substrate-replacement therapy developed to restore molybdenum cofactor synthesis and reduce neurotoxic metabolite accumulation in molybdenum cofactor deficiency Type A.
Services
End-to-end clinical trial services including protocol design, site selection, safety monitoring, natural history comparator studies, and study execution across phases.
Preparation and management of regulatory filings and health authority interactions, including pursuit of expedited regulatory designations and support for global submissions.
Support for commercial supply planning, demand/forecast integration, CMC project prioritization, and vendor/partner coordination to maintain uninterrupted supply for commercial products.
Development of payer-facing value materials, economic models, HTA support, and pre-approval information exchanges to enable reimbursement pathways.
Provision of no-charge genetic testing and counseling initiatives to identify eligible patients and support diagnosis for targeted genetic disorders.
Medical writing for clinical and regulatory documents, document QC and template/process ownership, and support for submission-ready dossier assembly.
End-to-end clinical trial services including protocol design, site selection, safety monitoring, natural history comparator studies, and study execution across phases.
Preparation and management of regulatory filings and health authority interactions, including pursuit of expedited regulatory designations and support for global submissions.
Support for commercial supply planning, demand/forecast integration, CMC project prioritization, and vendor/partner coordination to maintain uninterrupted supply for commercial products.
Development of payer-facing value materials, economic models, HTA support, and pre-approval information exchanges to enable reimbursement pathways.
Provision of no-charge genetic testing and counseling initiatives to identify eligible patients and support diagnosis for targeted genetic disorders.
Medical writing for clinical and regulatory documents, document QC and template/process ownership, and support for submission-ready dossier assembly.
Expertise Areas
- Clinical trial management
- Gene therapy development
- Small-molecule therapeutics
- Rare disease drug development
Key Technologies
- AAV9-based systemic gene delivery
- Substrate-replacement therapy
- Small-molecule negative allosteric modulators (GPCR-targeting)
- Genetic testing and gene-panel screening
News & Updates
Publication of positive clinical trial results for encaleret in NEJM.
Launch of Phase 3 clinical trial for encaleret targeting ADH1.
Publication of clinical data and study design announcement.
Published in The New England Journal of Medicine, detailing clinical trial results.
Results from clinical trials showing efficacy of encaleret.
FDA accepted the NDA for fosdenopterin, a treatment for MoCD Type A, with Priority Review.
Publication of positive clinical trial results for encaleret in NEJM.
Launch of Phase 3 clinical trial for encaleret targeting ADH1.
Publication of clinical data and study design announcement.
Published in The New England Journal of Medicine, detailing clinical trial results.
Results from clinical trials showing efficacy of encaleret.
FDA accepted the NDA for fosdenopterin, a treatment for MoCD Type A, with Priority Review.