As first announced in July 2019, Regenxbio Inc. is developing a one-time gene therapy candidate to deliver a gene encoding a therapeutic antibody against plasma kallikrein, a key protein of the plasma contact pathway which is left unregulated in patients with HAE.

Preclinical animal studies conducted using NAV AAV8 indicate the potential for a sustained and safe delivery of biologically active antibody at therapeutic concentrations. REGENXBIO expects to select a lead product candidate in the first half of 2020 and provide a program update in the second half of 2020.

REGENXBIO’s NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10.
(Source: Regenxbio)