Gemini Therapeutics has commenced patient enrolment in its Phase I study of GEM103 for the treatment of dry age-related macular degeneration (AMD).
Dry AMD is one of the common causes of blindness. There are currently no approved therapies to treat the condition.
The trial is a single ascending dose (SAD) clinical study to assess the safety, pharmacokinetics and pharmacodynamics of GEM103.
GEM103 is a recombinant, native Complement Factor H (CFH) protein developed to address dysregulation due to loss of CFH function variants in the eyes of the AMD patients.
Gemini CEO Jason Meyenburg said: “Gemini was founded to deliver precision medicines for eye disorders by combining genetic insights with clinical and biological expertise in complement.
“We are excited to take GEM103 into the clinic as a novel approach in complement modulation for a devastating disease with no therapeutic options.
“We look forward to topline data from this study and the potential to initiate a Phase II study, supported by data from our ongoing CLARITY natural history study examining the pathogenesis and progression of dry AMD in patients carrying CFH risk variants.”
The GEM103 study plans to enrol nine patients with dry AMD who have shown CFH mutations.
Study investigator Arshad Khanani said: “Clinicians are looking for breakthrough treatments to disrupt the pathology of dry AMD.
“The concept of treating genetically selected patients with GEM103 to potentially modulate and normalise the alternative complement pathway is an exciting approach.”
Based in Massachusetts, Gemini Therapeutics focuses on developing new treatments for AMD and other associated vision disorders.