Hit to Lead compound screening
DefiniGEN’s platform offers a series of in vitro human hepatocyte disease models to help support Hit to Lead compound screening initiatives
Triage therapeutic candidates
Our models are generated using a highly optimized parental donor which has been proven to display extraordinary differentiation characteristics which yield robust, reliable and reproducible hepatocytes after gene editing.
The result is a set of unique disease models with the same genetic background, but CRISPR gene edited to introduce a pathogenic mutation for the disease being investigated.
DefiniGEN’s scientific team are adept at analyzing a wide range of therapeutic modalities which have been proven to work reproducibly in these cell models including small molecules, AAV, lentiviral vectors, siRNA, antisense oligonucleotides, mRNA, plasmid and a variety of base editing therapeutic approaches.
Our screening approach
Our screening approach can help support assessment of efficacy of your candidates in a range of liver diseases.
OPTIMIZED ENDPOINT BIOASSAYS
RNAi therapeutics, small molecules, gene therapeutics
SKILLED ANALYSIS
We offer a complete solution to assess and triage your therapeutic candidates
CRISPR EXPERTISE
Unique disease models with the same genetic background
HIGHLY OPTIMIZED MODEL
Robust, reliable and reproducible hepatocytes after gene editing
Project workflow
01
Project consultation - Choose/develop a model
Our team of experts will structure a study according to your project specific requirements.
02
Select your assay and send us your compounds
Depending on your therapeutic modality, our team optimize treatment windows and dosing accordingly.
03
We work as a research partner
Offering bi-weekly updates and interim reporting.
Explore our models
Let’s work together.
Contact us to arrange a free consultation to discuss your project requirements today.