Trinity College Dublin-based Genable Technologies has secured €5m in financing to help progress its gene therapy technology to pioneer new therapies for the cure for retinitis pigmentosa, a disease that can lead to blindness.
The biopharmaceutical firm is developing new gene medicines to treat ‘dominant’ genetic diseases based on the work of Prof Jane Farrar, Dr Paul Kenna and Prof Peter Humphries at Trinity College Dublin (TCD).
Genable’s focus is on suppression/replacement gene therapy technology. The company said the funding will be used specifically to progress the development of its lead test product GT038 retinitis pigmentosa forward into clinical evaluation test trials on humans.
Those with rhodopsin-linked retinitis pigmentosa have a mutation in the rhodopsin gene, which causes their sight to worsen over time, eventually leading to blindness.
According to Genable, there are no available therapies for retinitis pigmentosa. Via GT038 – which was granted orphan drug designation by the European Medicines Agency in 2010 – the firm is pioneering a therapy to treat rhodopsin-linked retinitis pigmentosa that uses 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.
The founders said today this simple combination represents a new paradigm in medicine with the potential to cure this disease and not just treat the symptoms.
New investors Fountain Healthcare Partners alongside existing investors Delta Partners led today’s financing round.
Commenting on the new funding, Dr Ena Prosser, partner at Fountain Healthcare Partners, spoke about how the Trinity College-based team, along with the dedication and support of Irish families who carry genes that can lead to inheritable blindness, has pioneered research into this disease over several years.
“We believe that GT038 offers a significant technological breakthrough to address retinitis pigmentosa and we look forward to working closely with the company to accelerate the development of GT038 and other products to the market,” said Prosser.
Genable chairman Dr Geoffrey Vernon this morning thanked Delta Partners, Fighting Blindness Ireland, Science Foundation Ireland, Foundation Fighting Blindness-National Neurovision Research Institute (USA) and Enterprise Ireland for their ongoing support.
“We are extremely pleased to see GT038 raise the necessary finance to translate basic research performed at TCD into the clinic. It will help raise awareness of retinitis pigmentosa as a serious disease and ultimately help more patients receive therapy for their disease,” added Farrar, who is also co-founder of Genable Technologies.
In advance of Dublin City of Science 2012, Siliconrepublic.com is hosting Science November, a month dedicated to news, reports, interviews and videos covering a range of Irish science, research and innovation.