PTC299 is primarily investigated for its potential therapeutic applications in various diseases, particularly those involving abnormal blood vessel formation. It is being studied as a treatment for conditions like cancer, where it aims to inhibit angiogenesis, the process by which tumors develop new blood vessels to support their growth. By targeting this mechanism, PTC299 may help to starve tumors of essential nutrients and oxygen, potentially slowing their progression. Additionally, it is being explored for use in other angiogenesis-related disorders, such as certain eye diseases, where abnormal blood vessel growth can lead to vision loss. Research is ongoing to determine its efficacy and safety in these applications.