CTx001 for geographic atrophy: a gene therapy expressing soluble, truncated complement receptor 1 (mini-CR1)
Sonika Rathi, Athanasios Didangelos, Sofiya Pisarenka, Rachel Green, Stamatia Zafeiri, Peter G. Billcliff, Nakul Patel, Pamela Whalley, Parisa Zamiri, Viranga Tilakaratna, Ewa Szula, Rafiq Hasan, Mustafa M. Munye, Richard D. Unwin, Paul N. Bishop, , Simon J. Clark
This paper develops CTx001, a gene therapy expressing mini-CR1, which reduces MAC formation in a rat model of geographic atrophy.
Ophthalmology Science · Oct 22, 2025Peer-reviewedBiochemistry