NG101 Gene Therapy for Age-Related Macular Degeneration

This trial tests a new gene therapy called NG101 to determine its safety and effectiveness for individuals with wet age-related macular degeneration (wAMD), a condition that causes vision loss. The treatment involves a one-time injection into the eye, with three different dosage levels under evaluation. Ideal participants have been diagnosed with wAMD and have received anti-VEGF injections, a common treatment for this condition, in the past six months. The goal is to identify a potential new treatment that could improve or stabilize vision in those affected by wAMD. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this innovative therapy.

The trial requires that you stop taking any anticoagulant therapy (blood thinners) that is medically necessary at least 2 weeks before the NG101 injection, except for low-dose aspirin used for prevention.

Research has shown that NG101 gene therapy, tested for wet age-related macular degeneration (wAMD), has a good safety record in early trials. So far, no side effects directly linked to the drug have been reported, suggesting that people tolerate the treatment well. Some serious side effects have occurred, but these also appeared in patients who received a placebo (a treatment with no active ingredients), indicating they might not be related to NG101.

Before human testing, lab studies also supported the safety of NG101. These studies demonstrated that the treatment could be safe and effective. NG101 uses a virus to deliver a gene that helps block a protein called VEGF, which plays a role in wAMD. This method has been safe in early trials, giving researchers hope about its safety in people.

Researchers are excited about NG101 gene therapy for age-related macular degeneration because it offers a novel approach by directly targeting the genetic causes of the disease. Unlike standard treatments such as anti-VEGF injections that need frequent administration, NG101 is delivered through a single subretinal injection, potentially reducing the need for repeated treatments. Additionally, NG101 uses an adeno-associated virus (AAV) vector to deliver the gene therapy, which could provide longer-lasting benefits by addressing the root cause of vision loss. With different dose groups being studied, there's hope for optimizing its effectiveness and safety.

Research has shown that NG101 gene therapy could help treat wet age-related macular degeneration (wAMD). This therapy uses a modified virus to deliver a gene that enables the body to produce aflibercept, a protein that blocks VEGF, which causes harmful blood vessel growth in the eye. Studies have found that NG101 can be effective even at low doses, leading to a lasting presence of this beneficial protein. Early results suggest that this method might slow or improve vision loss in wAMD. While these findings are encouraging, more data from the various treatment arms in this trial, which test different doses of NG101, will provide a better understanding of its effectiveness.¹³⁴⁵⁶