Ultra-small Nucle­as­es

Ultra-small nucleases to expand in vivo delivery options; Our ultra-small nucle­as­es are high­ly effi­cient genome edit­ing sys­tems that have the poten­tial for exten­sive ther­a­peu­tic appli­ca­tions. We have pri­or­i­tized find­ing sys­tems to over­come the size con­straints of first gen­er­a­tion nucle­as­es. Our sys­tems are a frac­tion of the size and pack­age eas­i­ly into a sin­gle AAV for tar­get­ing organs beyond what is pos­si­ble with lipid nanopar­ti­cle delivery.