By integrating clinical, molecular, and genomic datasets, Nobias strives to craft a more precise and interconnected portrait of pathologic processes, potential therapeutic targets within pathologic processes, and optimal molecules and tools to modulate those therapeutic targets.
Led by a team of industry leaders in the fields of big data and artificial intelligence (AI), we use cutting-edge deep learning tools, clever algorithms, and a little bit of math to interrogate rich clinical data including genomic and molecular data sources.
These data sources include both public and private data. Our tools integrate them together with unique and proprietary access to one of the world’s largest and most diverse pediatric genomic datasets to drive new insights into human biology.
We leverage these integrations and these biological insights to drive accelerated development of novel therapeutics for patients who would otherwise have limited or minimal available treatment options.
At Nobias we have designed a suite of cutting-edge AI/ML-based drug discovery tools that span the gamut from early ideation, to insight development, to chemical development and optimization — individually polished and then cross-integrated into the Nobias WorkbenchTM.
We apply this proprietary, scalable, applied AI platform to unique data sets to unlock the secrets of disease biology.
Our insights into biological drivers of disease have already delivered two candidate breakthrough therapies for rare pediatric diseases.
22q11.2 Deletion Syndrome (22q11DS) is associated with delayed or incomplete development of several body structures or systems. The syndrome is also strongly associated with neuropsychiatric conditions, including inattention (ADHD), anxiety, autism, and schizophrenia/psychosis.
Nobias is developing an investigational metabotropic glutamate receptor (mGluR) modulator for some of the neuropsychiatric symptoms of 22q11DS. We have now completed enrollment of a multi-center, randomized, double-blind, placebo-controlled Phase 2 crossover trial. Results from our data analysis will be announced soon.
Vascular Anomalies (VAs) are a collection of rare, potentially life-threatening diseases caused by abnormal and/or dysregulated growth of arteries, veins, capillaries, and lymphatics, resulting in symptoms that significantly impact quality of life and may be fatal or life-threatening.
Nobias’ hospital partner discovered that mutations in a cellular stress response pathway led to overexpression of MEK in certain vascular anomaly patients. Recognizing the therapeutic role of MEK inhibition in the treatment of specific VAs (publication in Nature Medicine, July 2019), Nobias used tools from our Workbench to recapitulate these findings. After extensive testing and vetting using both in vitro/in vivo models and proprietary in-house in silico models, Nobias has selected a MEK inhibitor for further development. IND-enabling studies are ongoing.