Herein we investigated the potential of combining the complement regulatory properties of CD46, CD55 and CD59 into single gene products expressed from an adeno-associated virus (AAV) vector in a soluble non-membrane anchored form.

Jul 1, 2019 · This trial is studying the safety and tolerability of expressing CD59 from an adeno-associated virus (AAV) vector injected once into the vitreous. A second clinical trial assessing the efficacy of CD59 in wet AMD patients is also under way.

Aug 1, 2022 · Adeno-associated virus (AAV)-CAGsCD59 (Hemera Biosciences) is specifically designed to target the terminal phase of complement activation that can lead to the formation of MAC.

Jul 3, 2024 · Both AAV2/8-PEDF-P2A-sCD59 and AAV2/8-sFLT-1-P2A-sCD59 significantly reduced CNV development for an extended period without side effects and provided efficacy similar to two injections of current...

Oct 22, 2013 · We have previously described the development of a soluble membrane-independent form of CD59 (sCD59) that when delivered via a gene therapy approach using an adeno-associated virus vector (AAV2/8-sCD59) to the eyes of mice, can block MAC deposition and choroidal neovascularization.

Oct 20, 2017 · The complement system activation is involved in the development of anti‐neutrophil cytoplasmic antibody‐associated vasculitis (AAV). The study aimed to investigate the expression of complement regulatory proteins (CRPs) CD46, CD55 and CD59 in …

Jun 6, 2018 · Unexpectedly, AAV-mediated delivery of sCD59, an inhibitor of C9 incorporation into the MAC, successfully attenuated activation of the NLRP3 inflammasome and EAU pathology as well as MAC.

Apr 28, 2011 · Adeno-associated virus (AAV) serotype 2 has been shown to be safe when administered to the vitreous of non-human primates [23]. The sCD59 or GFP expression cassettes were cloned into an AAV-2 vector and are referred to as AAVCAGsCD59 and AAVCAGGFP respectively (Figure 7A).

In AMD, disease severity is correlated with the formation of the membrane attack complex (MAC), the terminal step in the complement cascade, as well as diminished RPE expression of CD59, a membrane-bound regulatory protein of MAC formation.

Herein we investigated the potential of combining the complement regulatory properties of CD46, CD55 and CD59 into single gene products expressed from an adeno-associated virus (AAV) vector in a soluble non-membrane anchored form.