Disease Model Pipeline

Indication

Model

Gene Targeting

Founders

Characterization

 

Heart Disease

ExeGen® LDLR-/-&+/-

     
 

Cystic Fibrosis

ExeGen® CFTR-/-

     
 
 

ExeGen® CFTRF508del/F508del

     
 

Cardiac Arrhythmia

ExeGen® SCN5A+/E558X

     
 

Cancer

ExeGen® TP53+/R167H, R167H/R167H

     
 
 

ExeGen® TP53+/R167H& KRAS+/G12D

    In Progress
 

Neuromuscular

ExeGen® DMD+/ex52del

     
 

Neurodegeneration

ExeGen® ATM-/-

     
 
 

ExeGen® CLN3ex7&8del/ex7&8del

    In Progress
 
 

ExeGen® HTT+/Ex1+(CAG)n

    In Progress
 

Hypertrophic Cardiomyopathy

ExeGen® MYH7+/R403Q

    In Progress
 

Nervous System

ExeGen® NF-1+/-

    In Progress
 
Renal Disease

ExeGen® PKD+/-

    In Progress
 
Blood Disorders

ExeGen® SCDhHBA/hHBA/hHBG-hHBBS/ hHBG-hHBBS