Interim results from a Phase II study by Gene Signal suggest that antisense oligonucleotide GS-101 is safe and effective at inhibiting abnormal corneal new blood vessel growth.

Published in Ophthalmology September 2009 Gene Signal is reported to be now conducting an international phase III trial with GS-101 for the prevention of pathologic corneal neovascularisation and thereby corneal graft rejection. GS-101 has been granted Orphan Drug status for this indication in Europe.

German researcher, Dr Claus Cursiefen of the Department of Ophthalmology, Friedrich-Alexander University Erlangen-Nürnberg told Medical News Today: "Compared to the placebo group in which 100% of patients suffered from progression of corneal neovascularisation, the optimal GS-101 treatment group showed regression in 86% of patients. We are very encouraged by these results as they represent real progress in the development of GS-101 as a new treatment to combat corneal graft rejection. We urgently need new options for the thousands of graft recipients, whose current treatment options for threatened rejection such as immunosuppressants are not ideal due to side effects. GS-101 is the first specific angiogenesis inhibitor that has demonstrated activity at the anterior part of the eye, where numerous diseases associated with pathologic angiogenesis endanger vision."