At Variant, we are developing breakthrough treatments to tackle unmet medical needs in rare genetic vision disorders.
Targeting the genetic root cause
VAR002 is our lead gene therapy candidate, designed to treat severe retinal diseases such as Leber congenital amaurosis, retinitis pigmentosa, and cone-rod dystrophy. These conditions are linked to mutations in the CRX gene, which is critical for the function of retinal photoreceptor cells.
Precise and efficient delivery technology
VAR002 uses a recombinant AAV5 vector to deliver a healthy copy of the CRX gene directly into cones and rods. Leveraging a photoreceptor-specific promoter (hGRK1), the therapy ensures targeted and localized expression of the CRX protein where it's needed most.
Local administration, long-term impact
Administered through a subretinal injection in a surgical setting, VAR002 acts at the molecular level to restore proper gene expression—aiming to slow, stabilize, or potentially reverse disease progression.
Our vision
To push the boundaries of ocular medicine by combining advanced biotechnology, genetic engineering, and translational design—bringing new hope to patients affected by rare inherited retinal disorders.