Frequently Asked Questions

What is Age-related Macular Degeneration (AMD)?

A progressive eye disease that causes blurred vision and eventually blindness. AMD is the most common cause of irreversible vision loss in people age 50 and older. With geographic atrophy (GA) due to AMD, loss of central vision is commonly observed due to deterioration of the back part of the eye, known as the retina. Risk for AMD increases in people over 60 years, family history of AMD, Caucasians and in smokers.

What is the GOLDEN study?

GOLDEN is a clinical study of an investigational antisense drug for the treatment of geographic atrophy (GA) due to age-related macular degeneration (AMD). The study will help determine the safety and effectiveness of the potential new treatment in slowing the progression of GA caused by AMD.

What differentiates the GOLDEN study from other trials related to GA or AMD?

Unlike other treatments meant to help people living with GA or AMD, the GOLDEN study is for an investigational drug that has the potential to treat both eyes through one monthly injection. And rather than an injection directly into an eye, the investigational drug is given through an injection just beneath the skin (subcutaneous) in either the abdomen or thigh. This means it has the potential of being more tolerable and more convenient than other treatments for this condition.

In addition, the investigational drug has a unique mechanism by targeting the genes shown to be responsible for the genetic causes of AMD.

What is the investigational drug used in the GOLDEN study?

The drug is based on antisense technology, which is a therapy for dramatically reducing disease-causing proteins. The study drug uniquely targets complement factor B, which is a protein found in the liver, blood and eyes, and a known risk factor for developing GA due to AMD. The study drug is designed to delay the progression of AMD by modulating or dampening complement factor B over-activity in the back of the eye.

How long is the GOLDEN study?

Individual participation in the GOLDEN study will take place over 18 months. This time consists of 3.5-month screening (known as the baseline period), an 11-month study treatment period, and a 3-month post-study treatment period.

What happens during the screening period?

During the screening period, you will be asked about your health and medical history. You’ll also have complimentary physical examinations, laboratory tests, ocular imaging performed and receive vaccinations, if needed, to make sure you are healthy enough and ready to participate.

Because this is a research study, if, after you’ve completed some or all of the examinations required during the screening period, you may or may not be eligible to participate in the study. We will discuss with you the reasons why.

Will everyone who participates in the study receive the study drug?

If you are eligible and agree to participate in this study, you will either receive the study drug or placebo during the study treatment period. A placebo is a substance that looks like and is taken the same way as the real drug but has no active ingredients or medical benefit.

In clinical trials, a placebo is given to some participants so that the two groups—for GOLDEN, it’s those taking the investigational drug vs. those taking the placebo—can be compared to show whether the drug being studied is safe and is effective. A placebo can also help researchers determine whether certain changes during the study happen by chance or actually in response to the investigational drug.

Additionally, participants receiving placebo should not be concerned about disease progression during the GOLDEN study because dry AMD is a slow-progressing disease and conditions are unlikely to worsen during the study treatment period.

What precautions are being taken to help protect the health of at-risk study volunteers?

During the GOLDEN study, your health is our number one priority. All medical guidelines are to be followed in study clinics including the use of gloves, properly preparing needles, and disposing of all materials. Masks, safety goggles or face shields may also be used for added protection.

How much will it cost to participate in the Golden Study?

There is no cost associated with participation in this study. Travel expenses (such as transportation and hotel, if required) will be provided. You may also be reimbursed for certain travel expenses that are related to your study visits (such as meals, parking, and taxi fares).

What is an antisense therapeutic?

An antisense therapeutic is an RNA-targeted therapeutic that targets the root cause of the disease and aims to reduce disease-causing proteins before they are even created.