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Precision Medicine Product Engine

Narrowing in on the cause of kidney disease opens a whole new world of treatment options.

 

Our precision medicine product engine is the foundation of our approach. Our product engine allows us to discover and validate novel targets aimed at understanding the molecular and phenotypic heterogeneity in diverse patient populations to identify subsets most likely to respond our treatments.

Kidney Genome Atlas

 

KGA™ Platform

Our KGA is a proprietary computational platform built in-house to identify genetic and environmental causes of kidney diseases in patient subsets and the associated biological targets for therapeutic intervention. Our KGA platform houses genomic, transcriptomic and clinical data from tens of thousands of people living with kidney disease. These data are mined using proprietary algorithms and other methods to identify molecular and environmental causes of kidney diseases.  Currently, the KGA platform consists of data from over 20,000 patients and population-based controls, and we continue to expand the data in the KGA into other kidney diseases.

Establishing a Worldwide Partner Network

To build our KGA platform, we have been establishing a large network of academic and commercial suppliers of clinical samples and phenotypic data from people with kidney disease.  Using a third-party vendor, we then sequence these samples to generate the genomic and transcriptomic data.  These data are then aggregated on a disease basis and analyzed in the KGA platform.  This supportive infrastructure of sourcing and sequencing patient samples can be leveraged as we continue to expand the KGA platform into other kidney diseases.

Human Organoid and Podocyte in vivo and in vitro Models

 

Our proprietary human stem cell-derived organoid and podocyte in vivo and in vitro models for target validation mimic human physiology and pathology narrowing the translational gap between preclinical models and clinical studies. We established a model in which we transplant organoids under the kidney capsule of small animal models, enabling maturation, and vascularization which facilitates in vivo evaluation, overcoming the previous challenges of not being able to undertake pharmacokinetic and pharmacodynamic analyses using organoids in vivo. Using our human organoids, we are enhancing our confidence in the translational relevance of novel targets and therapeutic agents. We believe our ability to recapitulate human kidney diseases with our transplanted human stem cell-derived organoids combined with the insights from our KGA platform will enable target identification and validation with an unprecedented level of translational confidence.

Small Molecule Drug Discovery Capabilities

 

We integrate novel tools and approaches with a strong team of industry experts in prosecuting a range of cytosolic and membrane bound targets including ion channels, GPCRs and kinases. We seek to improve the efficiency of drug discovery and time to clinical trials by integrating biology, lead discovery and chemistry during the later stages of target validation to accelerate hit finding. We then apply ligand and structure-based drug discovery approaches to establish structure activity relationships and to integrate multiparametric data which leads to novel insights and design hypotheses. Using a suite of computational, cheminformatics and molecular modeling tools, we can combine the cross functional data from structural biology and molecular modeling, in vitro, cellular, ADME, PK and in vivo experiments. Evidence of the success of our platform is the discovery campaign for GFB-887 which progressed from a high throughput screening hit to Phase 1 clinical trial in two years.