Atsena Therapeutics is developing gene therapies to prevent or cure blindness. The company has built a unique adeno-associated virus (AAV) technology platform to overcome common treatment challenges for inherited retinal diseases. The platform utilizes (1) laterally spreading capsids that ensure safe and widespread gene delivery to the retina and fovea, (2) dual vectors to deliver sufficient payload for mutations in large genes that cannot be treated with a single AAV vector, and (3) intravitreal capsids that are injected into the eye and optimized to avoid detection by the immune system, as patients often have pre-existing immunity to common AAVs.

<p>Atsena Therapeutics is developing gene therapies to prevent or cure blindness. The company has built a unique adeno-associated virus (AAV) technology platform to overcome common treatment challenges for inherited retinal diseases. The platform utilizes (1) laterally spreading capsids that ensure safe and widespread gene delivery to the retina and fovea, (2) dual vectors to deliver sufficient payload for mutations in large genes that cannot be treated with a single AAV vector, and (3) intravitreal capsids that are injected into the eye and optimized to avoid detection by the immune system, as patients often have pre-existing immunity to common AAVs.</p>


Atsena Therapeutics

Founded: 2020
Location: Durham, NC
Employee range: 11 - 50
Clinical stage: Clin1/Clin2
Therapy area: Leber congenital amaurosis 1
Drug types: OPH, GEN, OTO, RAR, PED
Lead product: ATSN-101
Funding: $55M A Dec 2020

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