Gene Therapies

OUR DEVELOPMENT OF GENE THERAPY

Almost all gene therapies to date have been developed for rare diseases. Gyroscope is developing gene therapy beyond rare disease to treat serious diseases of the eye. Our initial focus is advancing gene therapies for dry age-related macular degeneration (AMD), a leading cause of blindness.

Gyroscope’s lead investigational gene therapy, GT005, is currently being evaluated in Phase II clinical trials for the treatment of geographic atrophy (GA), the advanced form of dry AMD.1, 2 GT005 has received Fast Track designation from the U.S. Food and Drug Administration for the treatment of people with GA.3

GT005 aims to restore balance to an overactive complement system, a part of the immune system, by increasing production of the Complement Factor I (FI) protein, which regulates the activity of the complement system.4

Complement overactivation can lead to inflammation that damages healthy tissues, and it has been strongly correlated with the development and progression of AMD. It is believed that increasing FI production could reduce inflammation, with the goal of preserving a person’s eyesight.5

GT005 is designed to be a one-time treatment, and patients in our ongoing clinical trials receive a single administration of GT005 through an injection below their retina.1, 2

Find out more about our Clinical Trials

See our latest Presentations and Publications

REFERENCES

1. EXPLORE: A Phase II Study to Evaluate the Safety and Efficacy of Two Doses of GT005 (EXPLORE). ClinicalTrials.gov website. Updated December 2, 2021. Accessed November 14, 2022. https://clinicaltrials.gov/ct2/show/NCT04437368.

2. HORIZON: A Phase II Study to Evaluate the Safety and Efficacy of Two Doses of GT005. ClinicalTrials.gov website. Updated May 3, 2022. Accessed November 14, 2022. https://clinicaltrials.gov/ct2/show/NCT04566445.

3. Gyroscope Therapeutics Granted FDA Fast Track Designation for GT005, An Investigational Gene Therapy For Dry Age-Related Macular Degeneration. 2020. Available at: https://www.gyroscopetx.com/gyroscope-therapeutics-granted-fda-fast-track-designation-for-gt005-an-investigational-gene-therapy-for-dry-age-related-macular-degeneration/. Accessed November 14, 2022.

4. 2021 Retina Society presentation: Kiss S. Interim results from a first-in-human phase I/II gene therapy study (FOCUS) of GT005, an investigational AAV2 vector encoding complement factor I, in patients with geographic atrophy. Presented at Retina Society; Sept 30, 2021; Chicago, IL.

5. Lachmann PJ. The amplification loop of the complement pathways. Advances in Immunology. 2009;104:115-149.

REFERENCES

1. EXPLORE: A Phase II Study to Evaluate the Safety and Efficacy of Two Doses of GT005 (EXPLORE). ClinicalTrials.gov website. Updated December 2, 2021. Accessed November 14, 2022. https://clinicaltrials.gov/ct2/show/NCT04437368.

2. HORIZON: A Phase II Study to Evaluate the Safety and Efficacy of Two Doses of GT005. ClinicalTrials.gov website. Updated May 3, 2022. Accessed November 14, 2022. https://clinicaltrials.gov/ct2/show/NCT04566445.

3. Gyroscope Therapeutics Granted FDA Fast Track Designation for GT005, An Investigational Gene Therapy For Dry Age-Related Macular Degeneration. 2020. Available at: https://www.gyroscopetx.com/gyroscope-therapeutics-granted-fda-fast-track-designation-for-gt005-an-investigational-gene-therapy-for-dry-age-related-macular-degeneration/. Accessed November 14, 2022.

4. 2021 Retina Society presentation: Kiss S. Interim results from a first-in-human phase I/II gene therapy study (FOCUS) of GT005, an investigational AAV2 vector encoding complement factor I, in patients with geographic atrophy. Presented at Retina Society; Sept 30, 2021; Chicago, IL.

5. Lachmann PJ. The amplification loop of the complement pathways. Advances in Immunology. 2009;104:115-149.

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