Pioneering Gene Therapy for Geographic Atrophy, a Leading Cause of Vision Loss

Nadia Waheed MD, MPH, Chief Medical Officer, Gyroscope

September 30, 2021

As Chief Medical Officer of Gyroscope Therapeutics, a practicing retina specialist and a devoted researcher, I’ve spent my career working to preserve vision.

I’m particularly passionate about a leading cause of permanent vision loss for people over 55 called age-related macular degeneration (AMD).[1]

nadia waheed

The dry form of AMD accounts for 85-90% of cases.[2] As it advances it can lead to geographic atrophy (GA), where cells die in the retina causing loss of central vision.[3]

I’ve seen first-hand how GA steals people’s independence, ability to read, drive or even recognize the faces of loved ones. Currently, there are no approved treatments.

My ultimate goal, and the focus of all of us at Gyroscope, is to develop a one-time gene therapy designed to slow GA’s devastating progression.

Progress in Sight: Complement and AMD

This week, we are participating in the Retina Society’s Annual Scientific Meeting where research being shared reinforces a hypothesis that targeting the complement system can impact the progression of GA.

The complement system is an important part of the immune system, helping to defend against foreign intruders and clear away cell debris. However, research has shown that when it’s overactive, it can lead to inflammation that damages healthy tissues.[4]

It’s kind of like that old saying, “too much of anything is bad for you.”

Our Vision: Targeting Complement with Gene Therapy

Our approach uses gene therapy that is designed to transport a healthy gene into a cell to help the body make an important protein, in some cases to potentially compensate for a gene that is not working optimally.

Our investigational gene therapy, GT005, is designed to help the eye make more of a natural ‘regulatory’ protein called complement factor I (CFI). The role of CFI is to keep complement in check, and we are studying whether supplementing CFI in the eye can restore balance to an overactive complement system. 

Interim data from our ongoing Phase I/II study called “FOCUS” showed that the majority of GA patients treated with a single administration of GT005 had increased CFI levels in the vitreous (part of the eye), as well as decreases in proteins associated with activation of the complement system.

Two people in the study have nearly reached the two-year mark post treatment and their CFI levels remain increased from where they started, which is exciting to see.

From a safety perspective, GT005 has been well tolerated thus far with no treatment-related serious safety events.

While these results are early, they are encouraging. We are now enrolling two Phase II clinical trials that will help us understand if these positive effects translate into slowing disease progression.

Eye to the Future: Hope for the Field and the Patient Community

For me, Retina Society is an opportunity to reflect on the contributions made not only by our team but by everyone who is helping to move the science forward.  

For example, recent Phase III data for another company’s investigational complement inhibitor given monthly or bi-monthly for GA are also being presented, and while there is more to understand about the results, they are an important addition to the body of evidence that targeting complement may help combat the disease.

This is good news for all of us working to advance this field of research. At Gyroscope, it further supports our belief that restoring balance to the complement system with a one-time gene therapy has the potential to be a meaningful long-term solution.

Together, we have the potential to be the generation that brings forth the first treatments for people with dry AMD, whose courage inspires me every day.

[1] National Eye Institute. Age-Related Macular Degeneration. Page last reviewed June 22, 2021. Accessed September 28, 2021.

[2] American Macular Degeneration Foundation. What is Macular Degeneration? Accessed September 28, 2021.

[3] Bright Focus. What is Geographic Atrophy? Page last reviewed November 2020. Page last reviewed November 16, 2020. Accessed September 28, 2021.

[4] Garred, P. et al. (2021). Therapeutic Targeting of the Complement System: From Rare Diseases to Pandemics. Pharmacol Rev. 2021 Apr;73(2):792-827. doi: 10.1124/pharmrev.120.000072.