Nobias Therapeutics, Inc.
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)
Nobias Therapeutics, Inc.
3675 Market St., Suite 200, Philadelphia, PA 19104
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
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.
Execution and coordination of multicenter pediatric clinical trials including study design, site partnership, safety monitoring, and data analysis for rare-disease indications.
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.
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)
News & Updates
JPM Week
San Francisco, CA, January 13 – January 15, 2025
Sachs 8th Annual Neuroscience Innovation Forum
San Francisco, CA, January 12, 2025
Investival Showcase / London Life Sciences Week
London, UK, November 18, 2024
13th Biennial International 22q11.2 Scientific Meeting
Óbidos, Portugal, July 16 – July 18, 2024, where Nobias presented full results of Phase 2 study of NB-001 for the treatment of neuropsychiatric symptoms of 22q11DS.
BIO International Convention 2024
San Diego, CA, June 3 – June 6, 2024
Biotech Showcase 2024
San Francisco, CA, January 8 – January 10, 2024
JPM Week
San Francisco, CA, January 13 – January 15, 2025
Sachs 8th Annual Neuroscience Innovation Forum
San Francisco, CA, January 12, 2025
Investival Showcase / London Life Sciences Week
London, UK, November 18, 2024
13th Biennial International 22q11.2 Scientific Meeting
Óbidos, Portugal, July 16 – July 18, 2024, where Nobias presented full results of Phase 2 study of NB-001 for the treatment of neuropsychiatric symptoms of 22q11DS.
BIO International Convention 2024
San Diego, CA, June 3 – June 6, 2024
Biotech Showcase 2024
San Francisco, CA, January 8 – January 10, 2024