Our Approach
Scientific Approach
Scientific Approach

Our approach is guided by the specific needs
of each patient condition

ATSENA THERAPEUTICS’ platform is powered by adeno-associated virus (AAV) vectors engineered to overcome the unique hurdles presented by inherited retinal disease. The approach is guided by the specific needs of each patient condition. Our novel AAV gene therapy constructs are customized for ocular gene therapies. We utilize an AAV capsid, payload and delivery approach that is engineered for each disease, including dual vectors constructed to deliver larger genetic payloads for genetic mutations that have been untreatable with a single AAV vector.

Dual AAV vector approach for the delivery of MYO7A gene therapy

approach infographicBecause the MYO7A cDNA is too long to fit in a single vector, it is split in half and each half is delivered via a separate AAV vector. Upon co-infection of cells with both vectors, the gene halves recombine to form full length MYO7A.

Our capsid technologies are uniquely suited for the prevention or reversal of  blindness. We have designed novel adeno-associated virus (AAV) vectors for delivery via subretinal or intravitreal injection.

Uniquely suited
for the prevention or reversal of blindness

Our novel capsids for subretinal injection spread laterally well beyond subretinal injection site, delivering the therapeutic gene to a greater expanse of retina, which can increase the chance of a successful outcome. Notably, these capsids allow for transduction of 98% of foveal cones without the need for surgical detachment of the fovea. The unique features of these novel capsids allow for placement of vector in ‘safe’ areas of the retina and therapeutic gene expression in more fragile, distal regions.

AAVs are natural viruses that cause no harm to humans. Because a significant amount of people have been naturally exposed to them, patients often have pre-existing immunity to common AAVs that may prevent them from being candidates for trials or responding to therapy. This is especially important for intravitreally delivered gene therapies. Our novel AAV capsids for intravitreal injection are optimized and selected to avoid detection by patient immune system, expanding the potential patient population and use of therapy.

Our novel AAV capsid technology

approach infographic 2Capsids for intravitreal injection evade AAV neutralizing antibodies, are more potent than benchmark vectors and can be administered as an outpatient procedure.

Capsids for subretinal injection mediate transgene expression that spreads laterally across tissue, are highly potent and easy to manufacture.

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