RGX-381

RGX-381 is an investigational **AAV9-based gene therapy** being developed for the ocular manifestations of **CLN2 disease**, also known as late infantile Batten disease. The therapy is designed to deliver the **TPP1** gene to retinal cells through **subretinal administration**, with the goal of establishing durable intraocular expression of tripeptidyl peptidase 1 and slowing or preventing the progressive retinal degeneration, vision loss, and blindness associated with CLN2-related ocular disease. Originally developed by **REGENXBIO**, the program has also been referred to as **TTX-381** following a global licensing agreement with **Tern Therapeutics**.