Table of Contents
- What is ADENO-ASSOCIATED VIRUS SEROTYPE 2 ENCODING SOLUBLE CD59?
- How does it work?
- What condition does it treat?
- How is it administered?
- Current Clinical Trials
- Safety Monitoring
- Potential Benefits
- Important Considerations
What is ADENO-ASSOCIATED VIRUS SEROTYPE 2 ENCODING SOLUBLE CD59?
ADENO-ASSOCIATED VIRUS SEROTYPE 2 ENCODING SOLUBLE CD59 is an innovative gene therapy treatment currently being studied for the treatment of Geographic Atrophy (GA), a severe form of age-related macular degeneration (AMD). This therapy is also known by several other names, including HMR59, AAVCAGsCD59, JNJ-81201887, and JNJ-1887.[1]
How does it work?
This treatment uses a specially designed virus called an adeno-associated virus (AAV) to deliver a gene that produces a protein called CD59. Here’s a breakdown of how it works:
- The treatment uses a viral vector (a modified virus that can’t cause disease) to carry genetic material into cells.
- Specifically, it uses adeno-associated virus serotype 2 (AAV2), which is good at delivering genes to eye cells.
- The virus carries the instructions to make soluble CD59, a protein that can help protect cells in the eye.
- Once inside the eye cells, this genetic material instructs the cells to produce more CD59 protein.
- CD59 is believed to help protect eye cells from damage, potentially slowing down the progression of Geographic Atrophy.
This approach is a form of gene therapy, which aims to treat diseases by introducing genetic material into cells.[2]
What condition does it treat?
The primary target condition for this treatment is Geographic Atrophy (GA) secondary to Age-related Macular Degeneration (AMD). Let’s break down what this means:
- Age-related Macular Degeneration (AMD) is an eye disease that affects the central part of the retina, called the macula. The macula is responsible for sharp, detailed central vision.
- Geographic Atrophy (GA) is an advanced form of dry AMD. In GA, patches of cells in the retina die off, leading to blind spots in central vision.
- GA is called “geographic” because the areas of damage often look like a map to eye doctors when they examine the retina.
- This condition primarily affects older adults, typically those over 60 years of age.
Currently, there are no approved treatments to stop or reverse the progression of GA, which is why this new therapy is being studied with great interest.[1]
How is it administered?
ADENO-ASSOCIATED VIRUS SEROTYPE 2 ENCODING SOLUBLE CD59 is administered through an intravitreal injection. This means:
- The treatment is injected directly into the vitreous, the gel-like substance in the center of the eye.
- This is typically done in a doctor’s office or clinic setting.
- The injection is usually performed under local anesthesia to minimize discomfort.
- It’s a relatively quick procedure, often taking only a few minutes.
The treatment is provided as a solution for injection, and the dosage is measured in milliliters (ml).[1][2]
Current Clinical Trials
This treatment is currently being studied in clinical trials to evaluate its safety and effectiveness. Two notable trials are:
- Phase 2b Trial: This is a randomized, double-masked, multicenter study comparing intravitreal JNJ-81201887 to a sham procedure for treating GA secondary to AMD. The main goal is to measure the change in GA lesion growth in treated eyes compared to the control group.[1]
- Long-term Extension Study: This study is designed to monitor the long-term safety and tolerability of JNJ-81201887 in participants who received the treatment in previous clinical studies.[2]
Safety Monitoring
The long-term extension study is particularly focused on monitoring the safety of the treatment. This includes:
- Tracking any ocular (eye-related) and systemic (whole-body) side effects that may occur after treatment.
- Conducting regular clinical laboratory tests and safety assessments.
- Performing retinal imaging using techniques like fundus autofluorescence (FAF), spectral-domain optical coherence tomography (SD-OCT), and color fundus photography (CFP).
- Regular eye examinations to monitor any changes in eye health.[2]
Potential Benefits
While the full benefits of this treatment are still being studied, researchers hope it may:
- Slow down the growth of GA lesions in the eye.
- Help preserve central vision in people with GA.
- Potentially improve quality of life for people with this form of AMD.
However, it’s important to note that these potential benefits are still being evaluated in clinical trials.[1]
Important Considerations
If you’re considering participating in a clinical trial for this treatment, here are some important points to keep in mind:
- This is an investigational treatment, which means it hasn’t yet been approved for general use.
- Participation in clinical trials often involves regular follow-up visits and tests.
- There may be certain eligibility criteria, such as age restrictions (typically 60 years or older for these studies) and specific characteristics of your GA.
- As with any medical treatment, there may be potential risks and side effects, which the research team will discuss with you.
- The long-term effects of this gene therapy are still being studied.[1][2]
If you have Geographic Atrophy or are at risk for developing it, talk to your eye doctor about the latest treatment options and clinical trials that might be suitable for you.
