Retinal expression
AAV-IKV-mediated expression of a complement-regulatory prototype in retinal tissue.
Therapeutic program / Complement
Gene therapyC3-level regulation.
Terminal-pathway
protection.
Enhanced sCD59-based gene therapy combining upstream complement regulation with inhibition of membrane attack complex formation.
Scientific evidence
Demonstrated biological activity.
Company prototype data · mouse laser-CNV model and biochemical activity [1]
C3b inactivation
Retained Factor I-dependent cleavage of C3b, demonstrating upstream regulatory activity.
Reduced MAC deposition
Lower C5b-9 / membrane attack complex deposition in the mouse laser-CNV model.
Therapeutic approach
Building on prior sCD59 clinical experience.
An earlier sCD59 gene therapy was evaluated in human clinical studies [2]. Visiogene’s enhanced approach adds upstream C3/C3b regulation to terminal-pathway protection. The current program is distinct from the earlier clinically tested product.
- Therapeutic design
- C3/C3b regulation + terminal inhibition
- Disease focus
- Dry AMD / geographic atrophy
- Additional application
- Retinitis pigmentosa
Supporting sources
[1] Company prototype data. sCD59-based complement-regulation program.
Strategic partnerships
Start with the science.
Technology collaborations, strategic partnerships and investment discussions.