13 January 2020 - - US-based clinical stage precision medicine company Gemini Therapeutics has initiated enrollment in its Phase 1 study of GEM103, a recombinant, native Complement Factor H for the treatment of dry age-related macular degeneration, the company said.

AMD is the leading cause of irreversible blindness in the western world--affecting ms of people in the US alone. At least 90% of AMD patients have dry AMD, for which there are no approved therapies.

CFH risk variants are reported to occur in greater than 40% of patients with dry AMD and are strongly associated with the risk of developing the disease.

The complement system, of which CFH is a modulator, is dysregulated in patients with these risk variants and results in amplification of aberrant inflammatory responses in the eye. Over time this dysregulation leads to damage to the macular region of the retina.

GEM103 is a fully functional recombinant native CFH protein intended to address dysregulation caused by loss of CFH function variants in the eyes of these AMD patients.

This study is a Phase 1 single ascending dose clinical study evaluating the safety, pharmacokinetics and pharmacodynamics of GEM103. The study targets enrolment of approximately 9 patients with dry AMD who have known CFH mutations.

AMD is a progressive retinal disease affecting older adults, and the leading cause of irreversible blindness in the western world, affecting millions.

Symptoms, including blurry vision, loss of night vision and loss of central vision, make activities of daily living such as reading, driving and even recognizing faces more difficult over time. Dry AMD results from an interaction of both environmental and genetic risk factors.

Aging and smoking confer the strongest non-genetic risk, but genetic risk is significant, with approximately 70% attributable risk of advanced disease due to heritability.

Research over the last decade has uncovered multiple genetic variants which can increase risk of developing advanced AMD by up to 20-fold. Gemini Therapeutics is examining many of these genetic variants through CLARITY, its natural history study in people with dry AMD.

Gemini Therapeutics is a clinical stage precision medicine company developing innovative treatments for age-related macular degeneration and linked ocular disorders that are matched to molecular abnormalities found in patients with high clinical unmet need.

The company has generated a rich pipeline including recombinant proteins, monoclonal antibodies, and gene therapies. Gemini's CLARITY natural history study is designed to provide unprecedented insight into the role of genetic risk in common retinal diseases and began in December 2018.

Gemini was launched with funding from leading life science investors and powered by academic partnerships globally.