Ireland’s Genable Technologies Ltd secures Orphan Drug Designation by FDA for gene therapy product GT308

DUBLIN, Ireland, 12th April, 2013 - Genable Technologies Ltd gene therapy product, GT038, has been granted Orphan Drug Designation by the Federal Drug Administration (FDA) in the USA for the treatment of Retinitis Pigmentosa. This is a significant milestone as it provides Genable Technologies with seven years market exclusivity once GT308 has secured regulatory approval.

GT038 is an adeno-associated viral vector containing DNA encoding an RNAi targeting rhodopsin in combination with an adeno-associated viral vector containing a rhodopsin gene for the treatment of rhodopsin-linked Retinitis Pigmentosa. The product is currently undergoing formal pre-clinical assessments prior to commencing clinical studies in patients.

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. Almost 300,000 people worldwide have Retinitis Pigmentosa (RP) and of these, approximately 30% will have rhodopsin- linked (RP). 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 Alan Boyd MD of Genable Technologies Ltd said, ‘‘Retinitis Pigmentosa is a serious condition that leads to blindness in young people. There are currently no approved treatments for this condition and having orphan designation for GT038 in the US as well as Europe, is fundamental to the future development of our product that could have a significant impact on patients’ lives.’’

This orphan drug designation for GT038 by the FDA compliments the previous orphan drug designation granted by the European Commission to Genable Technologies Ltd, (Orphan Designation EU/3/10/817) for the treatment of rhodopsin-linked retinitis pigmentosa.


About Genable Technologies Ltd

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 company has secured financing from Fountain Healthcare Partners and Delta Partners. The background research has been supported by Fighting Blindness Ireland, Science Foundation Ireland, Foundation Fighting Blindness-National Neurovision Research Institute (USA), Enterprise Ireland & EVI-GenoRet (EU FP6-funded).


For additional information:

Eilish Joyce
FTI Consulting
Tel: +353 1 663 3609 / +353 87 791 4641