News
Genable Technologies to present at XVIIITH Annual congress of the European Society of Gene & Cell therapy
21st October 2010
Genable Technologies will present data on their novel approach for the treatment of RHODOPSIN-LINKED AUTOSOMAL DOMINANT RETINITIS PIGMENTOSA – a leading cause of blindness - at the XVIIITH ANNUAL CONGRESS OF THE EUROPEAN SOCIETY OF GENE & CELL THERAPY in Milan this weekend.
The use of gene-based medicines for many dominantly inherited diseases has been severely impeded by the presence of large numbers of unidentified disease causing mutations. Genable's novel "suppression and replacement" approach overcomes this serious impediment to finding a treatment for such diseases.
The lead drug candidate is delivered directly to the eye using adeno-associated virus (AAV). This novel therapy employs RNA interference (RNAi) - mediated suppression of rhodopsin in conjunction with the delivery of an RNAi-resistant replacement gene. Each component of the therapy, RNAi suppression and gene replacement, has now been shown to be functional in mouse models of the disease. Therapeutic benefit was demonstrated by using light and electron microscopy and electrophysiology and significant improvements in the vision of these mice was observed.
Dr Paul Kenna, the ophthamologist on the team commented "we have established robust proof of principle for this novel approach in a mouse model of retinitis pigmentosa. The therapy shows great promise for the treatment of RP patients as does the approach for other dominantly inherited diseases and we are looking forward to advancing towards a clinical study."
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, The Wellcome Trust, Foundation Fighting Blindness-National Neurovision Research Institute (USA), Enterprise Ireland & EVI-GenoRet.
