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Related Experiment Video

Updated: Jan 9, 2026

High-resolution Melting PCR for Complement Receptor 1 Length Polymorphism Genotyping: An Innovative Tool for Alzheimer's Disease Gene Susceptibility Assessment

High-resolution Melting PCR for Complement Receptor 1 Length Polymorphism Genotyping: An Innovative Tool for Alzheimer's Disease Gene Susceptibility Assessment

Published on: July 18, 2017

CTx001 for Geographic Atrophy: A Gene Therapy Expressing Soluble, Truncated Complement Receptor 1 (Mini-CR1).

Sonika Rathi^1,2, Athanasios Didangelos³, Sofiya Pisarenka³

¹Institute for Ophthalmic Research, Eberhard Karls University of Tübingen, Tübingen, Germany.

Ophthalmology Science

|December 11, 2025

Summary

This summary is machine-generated.

This study shows that CTx001 gene therapy effectively inhibits complement overactivation, a key factor in geographic atrophy (GA). The therapy delivers mini-CR1 protein to the retina, supporting its potential as a one-time treatment for GA.

Keywords:

AAV AMD Complement Gene therapy Macular degeneration

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Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium

Published on: August 7, 2015

Area of Science:

Ophthalmology
Gene Therapy
Complement System Biology

Background:

Geographic atrophy (GA) is an advanced form of age-related macular degeneration.
Complement system overactivation is implicated in the pathogenesis of GA.
Current treatments for GA are limited, highlighting the need for novel therapeutic strategies.

Purpose of the Study:

To conduct a preclinical evaluation of CTx001, a novel gene therapy for geographic atrophy (GA).
To assess the efficacy of CTx001 in delivering mini-CR1 (a complement receptor 1 fragment) to ocular tissues.
To investigate the complement-modulating activity of CTx001 in vitro and in vivo.

Main Methods:

CTx001, a self-complementary rAAV2 vector expressing mini-CR1, was evaluated.
In vitro studies included complement degradation assays, Wieslab assays, and mini-CR1 binding kinetics.
Ex vivo and in vivo assessments involved evaluating mini-CR1 diffusion across Bruch's membrane and subretinal administration in a rat model of choroidal neovascularization (CNV).

Main Results:

Mini-CR1 demonstrated potent cofactor activity, inhibiting both alternative and classical complement pathways with an IC50 of 125 nM.
CTx001-transduced RPE cells secreted mini-CR1, which diffused across Bruch's membrane and retained activity.
Subretinal administration of CTx001 in rats reduced MAC deposition in CNV lesions by 75.4%.

Conclusions:

CTx001 is a potent inhibitor of complement activation.
The gene therapy efficiently transduces RPE cells and delivers functional mini-CR1 to ocular tissues.
These findings support CTx001's further development as a one-time gene therapy for GA.