DISCOVERING AND DEVELOPING
Gyroscope was founded to explore the convergence of advancements made in the understanding of the complement system’s impact on eye disease, the genetic basis of age-related macular degeneration (AMD) and gene therapy as a mode of sustained treatment delivery.
Retinal Disease
Retinal diseases, like dry age-related macular degeneration, affect a person’s ability to see and can cause blindness.
The Retina
The retina is a thin layer of tissue inside the back wall of the eye that contains millions of light-sensitive cells and other nerve cells that receive and organize visual information.
Your retina sends this information to your brain through your optic nerve, enabling you to see.
While retinal disease can vary widely, they share some common signs and symptoms including:1
- Seeing floating specks or cobwebs
- Blurred or wavy vision
- Defect in the side vision
- Loss of vision
Dry Age-Related Macular Degeneration
Dry age-related macular degeneration (AMD), a retinal disease, is a leading cause of permanent vision impairment for people aged 50 and older and there are no approved treatments.
It is an incurable eye disease that steals your central vision over time and can eventually leave you blind.
You may lose the ability to read, drive a car or even recognize faces. And your greatest risk for getting this disease is simply growing old.2, 3
Gyroscope is committed to
FIGHTING THE DEVASTATING
IMPACT OF BLINDNESS
complement system
Research suggests that when a part of the immune system, the complement system, is overactive it leads to inflammation that damages healthy eye tissues.
The Complement System
The complement system is a collection of proteins inside your body that, when activated, enhance or “complement” your immune system.
It plays a key role in our body’s natural defense against infections, helping us clear away things that shouldn’t be there.4
Research has shown that an overactive complement system can lead to unnecessary inflammation that damages healthy eye tissue, causing vision loss.5
Complement Factor I
The late Sir Peter Lachmann first identified a key regulator that helps keep the complement system in check. It is a protein called Complement Factor I, or FI.6
Based on this finding, we created an investigational gene therapy encoding the transgene for human FI which is designed to make patient cells produce more FI protein.
Our theory is that the FI protein created can then naturally dampen, or slow down, the overactivity of the complement system and preserve a person’s eyesight.
Genetics
Genetics play an important role in determining the risk of developing dry age-related macular degeneration.
Genetics is the study of inheritance and understanding what different qualities or traits are passed down from your parents to you. Specific variations in your genetic makeup could suggest that you have an increased likelihood of developing a particular disease.7
Multiple independent genome-wide association studies, or GWAS, have confirmed that genetics play a major role in determining the risk of developing dry age-related macular degeneration (AMD) – many of the genes identified encode components of the complement system.8
A study by David Kavanagh, one of the scientific founders of Gyroscope, identified a group of people who had rare changes (called genetic variants) in the CFI gene.9
Most importantly, people with advanced dry AMD who had a rare FI gene variant also had lower average FI levels.
This finding was critical in designing our clinical development program, informing how we identify and stratify people in our clinical studies.
Gene Therapy
One-time gene therapies may provide durable and lasting benefit.
Genes and Your Health
Your genes play an important role in your health. They contain the information needed to make proteins, which support the structure, function and regulation of systems, tissues and organs within the body to keep you healthy.7
Gene Therapy
Gene therapy is a type of medicine that uses a carrier, called a vector, to deliver a functioning gene to specific cells in your body.10 The goal is to supplement working genes or replace any malfunctioning gene to help your cells produce proteins needed to naturally alleviate a disease or medical condition.11
Gene Therapy and Dry AMD
Almost all gene therapies to date have been developed for rare genetic diseases. Gyroscope is developing gene therapy beyond rare disease to treat a leading cause of blindness, dry age-related macular degeneration (AMD).
Typically, medicines used to treat retinal diseases are given through an injection in the eye as frequently as once a month. One of the key differences between gene therapy and other types of medicines is that gene therapies are designed to be one-time therapies that will hopefully provide years of benefit.12
Patients in our ongoing Phase I/II clinical trial receive a single dose of our investigational gene therapy through an injection below their retina. The goal is to slow the progression of dry AMD.
REFERENCES
1. Retinal Diseases. Mayo Clinic. Published January 6, 2022. Accessed November 14, 2022. https://www.mayoclinic.org/diseases-conditions/retinal-diseases/symptoms-causes/syc-20355825.
2. Gehrs KM, et al. Ann Med. 2006;38(7):450-471.
3. What is AMD? Prevent Blindness. Published February 2, 2022. Accessed November 14, 2022. https://preventblindness.org/amd-age-related-macular-degeneration/.
4. Gehrs KM, et al. Arch Ophthalmol. 2010;128(3):349-358.
5. Lachmann PJ. The amplification loop of the complement pathways. Advances in Immunology. 2009;104:115-149.
6. Lachmann PJ. Advances in Immunology. 2009;104:115-149.
7. What is Genetics? National Institute of General Medical Sciences. Published May 4, 2022. Accessed November 14, 2022. https://nigms.nih.gov/education/fact-sheets/Pages/genetics.aspx.
8. Fleckenstein M, et al. Ophthalmol. 2018;125(3):369-390.
9. Kavanagh D, et al. Hum Mol Genet. 2015;24(13):3861-3870.
10. Trapani I, et al. Prog Retin Eye Res. 2014;43:108-128.
11. What is Gene Therapy? U.S. Food and Drug Administration. Published July 25, 2018. Accessed November 14, 2022. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/what-gene-therapy.
12. Goswami R, et al. Front Oncol. 2019;9:297.
REFERENCES
1. Retinal Diseases. Mayo Clinic. Published January 6, 2022. Accessed November 14, 2022. https://www.mayoclinic.org/diseases-conditions/retinal-diseases/symptoms-causes/syc-20355825.
2. Gehrs KM, et al. Ann Med. 2006;38(7):450-471.
3. What is AMD? Prevent Blindness. Published February 2, 2022. Accessed November 14, 2022. https://preventblindness.org/amd-age-related-macular-degeneration/.
4. Gehrs KM, et al. Arch Ophthalmol. 2010;128(3):349-358.
5. Lachmann PJ. The amplification loop of the complement pathways. Advances in Immunology. 2009;104:115-149.
6. Lachmann PJ. Advances in Immunology. 2009;104:115-149.
7. What is Genetics? National Institute of General Medical Sciences. Published May 4, 2022. Accessed November 14, 2022. https://nigms.nih.gov/education/fact-sheets/Pages/genetics.aspx.
8. Fleckenstein M, et al. Ophthalmol. 2018;125(3):369-390.
9. Kavanagh D, et al. Hum Mol Genet. 2015;24(13):3861-3870.
10. Trapani I, et al. Prog Retin Eye Res. 2014;43:108-128.
11. What is Gene Therapy? U.S. Food and Drug Administration. Published July 25, 2018. Accessed November 14, 2022. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/what-gene-therapy.
12. Goswami R, et al. Front Oncol. 2019;9:297.
