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.

DefiniGEN Laboratory
DefiniGEN Scientist

Our screening approach

Our screening approach can help support assessment of efficacy of your candidates in a range of liver diseases.
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OPTIMIZED ENDPOINT BIOASSAYS

RNAi therapeutics, small molecules, gene therapeutics

 

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SKILLED ANALYSIS

We offer a complete solution to assess and triage your therapeutic candidates

 

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CRISPR EXPERTISE

Unique disease models with the same genetic background

 

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HIGHLY OPTIMIZED MODEL

Robust, reliable and reproducible hepatocytes after gene editing

 

Project workflow 

Explore our models

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Let’s work together.

Contact us to arrange a free consultation to discuss your project requirements today.