CTx001 / Complement Therap - LARVOL DELTA

CTx001 for Geographic Atrophy: A Gene Therapy Expressing Soluble, Truncated Complement Receptor 1 (Mini-CR1). (PubMed, Ophthalmol Sci) - "These findings support its further development as a 1-time gene therapy for addressing complement overactivation in GA. Proprietary or commercial disclosure may be found in the Footnotes and Disclosures at the end of this article."

Journal • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Gene Therapies • Macular Degeneration • Ophthalmology • Retinal Disorders • CR1

Nonclinical evaluation of CTx001, a gene therapy for the treatment of geographic atrophy in aged-related macular degeneration (ESGCT 2024) - "In this eight week, non-GLP dose range finding study, CTx001 produced at clinical scale maintained bioactivity in vivo and was well tolerated following subretinal administration in NHP up to maximal feasible dose. These results support initiation of IND enabling GLP toxicology studies for CTx001."

Gene therapy • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Gene Therapies • Hematological Disorders • Inflammation • Macular Degeneration • Ocular Inflammation • Ophthalmology • Retinal Disorders

Impact of cell line selection on AAV2 viral vector genome packaging efficiency (ESGCT 2024) - "CTx001 is an AAV2 gene therapy encoding a novel soluble and potent complement regulatory protein, mini-CR1, for the treatment of Geographic Atrophy. This reduction was corroborated using a number of analytical techniques. These results demonstrate the substantial impact the choice of cell line can have on AAV viral vector genome packaging efficiency."

Preclinical • Viral vector • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Gene Therapies

The FDA has cleared Complement Therapeutics GmbH’s (CTx) investigational new drug (IND) application for CTx001, its lead gene therapy candidate under clinical development to treat geographic atrophy secondary to age-related macular degeneration (AMD). (Eyes On Eyecare) - "The FDA’s IND clearance paves the way for CTx to conduct an international first-in-human (FIH), open-label, phase 1/2 clinical trial."

IND • New P1/2 trial • Age-related Macular Degeneration

CTx001 an AAV2 Mini-CR1 Gene Therapy for dry AMD: IND-Enabling Studies Show Potent Complement Target Engagement and MAC Inhibition on Activated RPE Cells (ESGCT 2025) - No abstract available

Gene therapy • Dry Age-related Macular Degeneration • Gene Therapies • CR1

CTx001, an AAV2 complement inhibitor gene therapy in nonclinical development for dry AMD, attenuates alternative and classical complement pathway activation in vitro and in vivo (ARVO 2025) - "Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

Gene therapy • Preclinical • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders • CR1