A WORLD LEADER IN RIBOSOME TARGETED GENETIC THERAPIES

DEEP PIPELINE of
First-in-class therapies

Our Pipeline

 
 
 
 
 
 
 
Indication
Target
Discovery
/Optimize
IND
Phase I
Phase II
Phase III(Pivotal)
Alport Syndrome (nonsense)
Collagen IV
ELX-02 (SC)
 
 
 
 
 
 
 
 
 
RDEB/JEB (nonsense)
Collagen VII/LAMB3
ZKN013 (oral)
 
 
 
FAP
(nonsense)
APC
ZKN013 (oral)
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 

Class I CF: Cystic fibrosis patients with nonsense mutations; RDEB/JEB: Recessive Dystrophic/Junctional Epidermolysis Bullosa;FAP: Familial adenomatous polyposis

Learn more about our programs for: