A TRINITY College-based genetics firm has raised €5 million to bring what it hopes will be a breakthrough treatment for a form of blindness to clinical trials.
The academics behind Genable Technologies have spent over 20 years looking at one form of inherited blindness – retinitis pigmentosa (RP) – in which a genetic mutation leads to damage of the retina and a gradual loss of sight.
Focusing on dominant genetic diseases, where a defective gene in one parent “dominates” the other parent’s normal gene in their child, the team has developed a “suppression and replacement” therapy.
A single injection to the eye will suppress the mutated gene and replace it with a healthy version of the gene, genetically modified to ensure it cannot, in turn, be suppressed. The treatment holds the potential to provide a first cure for as many as 30,000 patients in the US and Europe alone, a potential market Genable estimates at over $500 million.
The treatment involves a once-off injection in the eye area, using a virus known to be well tolerated in that area.
Having succeeded in establishing proof of principle for the drug, GT038, the company is now moving towards human trials to prove the safety and efficacy of the treatment.
Geneticist Prof Jane Farrar – one of the team with Prof Peter Humphries and opthamologist Dr Paul Kenna – said: “We are looking from a patient perspective to get something that works, a potent and efficacious therapy for patients. There is nothing on the market.”
She hailed post-doctoral scientists at Trinity as the “engine” in developing the revolutionary approach to the disease.
The fundraising, led by Fountain Healthcare Partners and also involving existing investor Delta Partners, will provide the funds to drive those trials.
“Opthamology has been one of the stars of of venture capital investment in the past year or two,” said Ena Prosser , partner at Fountain Healthcare. “It’s a very hot area and given we have one [prospect] under our nose in Dublin, it makes sense to get involved. It is a very reputable area to have in our portfolio of investments . . . We think it is primarily a first in class approach in the ocular area.”
Dr Prosser said the company would eventually need to consider licensing its technology or consider a sale at some later stage to support larger late stage clinical trials, if initial trials deliver on expectations.
Welcoming the involvement of Fountain Healthcare and Delta in funding the firm’s future development, Genable chairman Geoffrey Vernon also noted the ongoing commitment of charity Fighting Blindness Ireland, the Foundation for Fighting Blindness-National Neurovision Research Institute (USA) and Enterprise Ireland.
