Re-dosable, titratable genetic medicines

Liver disorders

We are advancing a diverse portfolio of therapeutic candidates, formulated in lipid nanoparticles, for diseases of the liver.

Liver disorders

GSD1a

Hemophilia A

PFIC

PKU

Eye disorders

Our ceDNA technology enables a broad portfolio of ophthalmology candidates, which can be delivered to the eye using multiple formulations.

Eye disorders

LCA10

Stargardt

Exploratory areas

We have also demonstrated durable in vivo expression in two additional tissues: the muscle and the central nervous system. In addition, we continue to work to unlock tractable pharmacology in several other important tissues.

  • MuscleMuscle
  • CNSCNS
bottom image