Nobias Therapeutics
Nobias Therapeutics is a clinical-stage biotechnology company focused on developing novel small-molecule therapeutics for rare pediatric diseases. Leveraging rich genomic and health datasets along with strong relationships with pediatric academic medical centers across North America, the company aims to rapidly advance drug discovery for genetically-defined rare diseases, initially targeting pediatric indications with potential expansion to adults and non-rare conditions.
Industries
Nr. of Employees
small (1-50)
Products
Orally dosed small-molecule mGluR activator for neuropsychiatric symptoms in 22q11.2 deletion syndrome (Phase 2 completed)
A development-stage, orally administered small molecule that activates multiple metabotropic glutamate receptors, evaluated in a randomized, placebo-controlled Phase 2 trial in children with neuropsychiatric symptoms associated with 22q11.2 deletion syndrome; demonstrated safety, tolerability, and positive responder-rate trends.
Small-molecule MEK inhibitor candidate for Ras/MAPK-driven complex lymphatic anomalies (IND-enabling)
A development candidate selected for IND-enabling studies as a MEK inhibitor intended to treat complex lymphatic anomalies caused by mutations in the Ras/MAPK pathway; selection informed by comparative screening in zebrafish models.
Orally dosed small-molecule mGluR activator for neuropsychiatric symptoms in 22q11.2 deletion syndrome (Phase 2 completed)
A development-stage, orally administered small molecule that activates multiple metabotropic glutamate receptors, evaluated in a randomized, placebo-controlled Phase 2 trial in children with neuropsychiatric symptoms associated with 22q11.2 deletion syndrome; demonstrated safety, tolerability, and positive responder-rate trends.
Small-molecule MEK inhibitor candidate for Ras/MAPK-driven complex lymphatic anomalies (IND-enabling)
A development candidate selected for IND-enabling studies as a MEK inhibitor intended to treat complex lymphatic anomalies caused by mutations in the Ras/MAPK pathway; selection informed by comparative screening in zebrafish models.
Services
Collaborative drug discovery using genomic and clinical datasets
Partner-based discovery using integrated pediatric genomic and EHR datasets combined with applied AI to identify disease drivers and therapeutic targets for genetically-defined rare diseases.
Clinical development and multi-center trial coordination for pediatric rare diseases
Execution and coordination of multicenter pediatric clinical trials including study design, site partnership, safety monitoring, and data analysis for rare-disease indications.
Collaborative drug discovery using genomic and clinical datasets
Partner-based discovery using integrated pediatric genomic and EHR datasets combined with applied AI to identify disease drivers and therapeutic targets for genetically-defined rare diseases.
Clinical development and multi-center trial coordination for pediatric rare diseases
Execution and coordination of multicenter pediatric clinical trials including study design, site partnership, safety monitoring, and data analysis for rare-disease indications.
Expertise Areas
- Clinical trial management
- Pediatric rare disease therapeutics
- AI-driven deep phenotyping
- Small-molecule drug discovery
Key Technologies
- AI-based deep phenotyping
- EHR-genomic data integration
- In vivo zebrafish screening models
- MEK inhibition (small-molecule pharmacology)