News

GT083 Granted orphan drug designation by the European Medicines Agency

31st January 2010

On 17th December 2010 orphan designation (EU/0/00/000) was granted by the European Medicines Agency (EMA) to Genable Technologies Ltd, Ireland, for GT038, an adeno-associated viral (AAV) based therapy containing DNA encoding an RNAi targeting rhodopsin and a replacement rhodopsin gene for the treatment of rhodopsin-linked Retinitis Pigmentosa.

Click here to read the announcement on the European Medicines Agency website

Patients with rhodopsin-linked Retinitis Pigmentosa have a mutation in the rhodopsin gene which causes a patient's sight to worsen, eventually leading to blindness. GT038 is a novel and unique therapy for rhodopsin-linked Retinitis Pigmentosa that utilizes AAV vectors to obtain expression of RNA interference molecules which suppress the expression of the faulty gene and replaces this with a gene encoding a functioning protein. This simple combination overcomes the significant hurdle in diseases such as rhodopsin-linked Retinitis Pigmentosa of mutation variability by eliminating the need to target specific mutations.

Professor Jane Farrar of Trinity College Dublin and Genable Technologies Limited commented "We are extremely pleased to see GT038 receive orphan drug status in Europe. This decision by the EMA will help raise awareness of of Retinitis Pigmentosa as a serious disease and ultimately help more patients receive therapy for their disease".

Genable Technologies Ltd. is a privately held, venture backed, Dublin (Ireland) based bio-pharmaceutical company developing new gene medicines to treat "dominant" genetic diseases based on the pioneering work of Professor Jane Farrar, Dr Paul Kenna & Professor Peter Humphries of Trinity College Dublin. The background research has been supported by Fighting Blindness Ireland, Science Foundation Ireland, Foundation Fighting Blindness-National Neurovision Research Institute (USA), Enterprise Ireland & EviGenoRet.