Gene Signal is currently developing aganirsen GS-101 (an antisense oligonucleotide or short naturally occurring DNA strand). GS-101 is a topical medication in the form of eye drops, and is currently being researched for the management of excessive vascular growth in the cornea, also known as “neovascularisation”
GS-101 is a copy of a naturally occurring human gene and acts by inhibiting the expression of IRS-1, a protein required for the formation and growth of new blood vessels. By blocking the expression of this protein in pro-angiogenic conditions (in this case, those with excessive vascular growth), GS-101 inhibits and regresses corneal neovascularisation, which interferes with vision and may be the cause of graft rejection.
Phase I, Phase II and Phase III clinical studies have been completed for Gene Signal’s GS-101 in the management of Corneal Graft Rejection associated with Neovascularisation (excessive vascular growth of the cornea).
The Phase I trial evaluated the safety of GS-101 is healthy male volunteers – which is what phase I studies are for. During Phase II trials, a drug is typically tested for safety and efficacy in a larger numbers of people who are actually afflicted with the disease or condition for which the drug is being developed. Gene Signal has also completed this step. Most recently, Gene Signal has also completed its Phase III I-CAN Study which demonstrated that topical applications of aganirsen are safe and well-tolerated, that they reduce the relative area of corneal neovascularisation and need for corneal transplantation.