Complement Therapeutics Ltd (CTx), a preclinical stage biotechnology company, has enrolled the first patient in a new study of geographic atrophy (GA) secondary to age-related macular degeneration (dry AMD).
Tell me about this study.
The investigating Geographic Atrophy Insights (i-GAIN) study is a multicenter, prospective, 12-month natural history study expected to enroll 250 patients across the United Kingdom with a confirmed diagnosis of GA.
As a non-interventional study, i-GAIN will assess enrollees’ status at baseline, 6 months, and 12 months. No experimental or investigational treatments will be provided.
What data will be analyzed?
Researchers will collect data on genetics, blood biomarkers, and phenotypic changes in patients’ eyes. The findings will support the development of CTx001, CTx’s lead gene therapy investigational candidate for complement-driven AMD.
Tell me more about CTx001.
The company’s lead asset is an adeno-associated virus (AAV)-based gene therapy currently being studied in CTx’s development program for GA. Future programs are expected to evaluate the therapeutic potential for CTx001 to treat other complement-mediated conditions.
Significance?
With no treatments currently approved for GA, Marta Ugarte, DPhil, FRCOphth, principal investigator of the clinical development program, said that the study will enable “researchers to gain a better understanding of the disease” and “provide insights into how the [GA] progresses.”