DISCOVERING, DEVELOPING AND DELIVERING
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 diseases, like dry age-related macular degeneration, affect a person’s ability to see and can cause blindness.
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:
- 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 the leading cause of permanent vision impairment for people aged 65 and older and there are no approved treatments.
It is an incurable eye disease that steals your central vision overtime and can eventually leave you blind.
You lose the ability to read, drive a car or even recognize faces. And your greatest risk for getting this disease is simply growing old.
Gyroscope is committed to
FIGHTING THE DEVASTATING
IMPACT OF BLINDNESS
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.
Research has shown that an overactive complement system can lead to unnecessary inflammation that damages healthy eye tissue, causing vision loss.
Complement Factor I
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 CFI.
Based on this finding, we created a gene therapy that delivers a copy of the CFI gene into a person’s cells to create more CFI protein.
Our theory is that the CFI protein created can then naturally dampen, or slow down, the overactivity of the complement system and preserve a person’s eyesight.
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.
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.
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.
Most importantly, people with advanced dry AMD who had a rare CFI gene variant also had lower average Factor I levels.
This finding was critical in designing our clinical development program, informing how we identify and stratify people in our clinical studies.
One-time gene therapies may provide durable and lasting benefit.
Genes and Your Health
Your genes play an important role in keeping you healthy. If a gene is defective and not acting in the way that it should, it can change how important proteins in your body are made which can ultimately lead to disease.
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. The goal is to replace any malfunctioning genes or supplement working genes so that your cells can produce the proteins needed to naturally alleviate the disease or medical condition.
Gene Therapy and Dry AMD
Almost all gene therapies to date have been develop for specific and rare genetic diseases. Gyroscope is developing gene therapy beyond rare disease to treat a leading cause of blindness, dry age-related macular degeneration (dry-AMD).
Typically, medicines use 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 one-time therapies that will hopefully provide years of benefit.
Patients in our ongoing Phase I/II clinical trial, known as the FOCUS study, receive a single dose of our therapy through an injection below their retina. The goal is to slow, or possibly even stop the progression of dry-AMD.