By Cormac Sheridan
BioWorld International Correspondent
DUBLIN, Ireland ¿ One of Ireland¿s most successful life sciences research groups is spinning off a start-up company, Optigen Technologies Ltd., to develop therapeutic strategies for treating dominant genetic diseases.
The ocular genetics research unit, based at the Smurfit Genetics Institute at Trinity College Dublin (TCD), has built up a strong track record over the past 15 years in the molecular genetics of retinitis pigmentosa (RP), a set of inherited degenerative conditions affecting the retina that lead eventually to blindness. It was at the forefront of efforts to map and characterize autosomal dominant forms of RP in the early 1990s. Since then, it has developed and patented a technology platform that is broadly applicable to the suppression of dominant genetic mutations.
¿Recessive disease is much easier to treat,¿ said Optigen¿s acting CEO, Jane Farrar, who, along with Peter Humphries and Paul Kenna, is co-director of the TCD ocular genetics group. Such conditions need the addition of a working wild-type allele, whereas dominant mutations give rise to the production of proteins with harmful physiological effects. Over 1,000 dominant conditions have been described in humans, Farrar said.
The nascent company will itself concentrate on three areas ¿ RP, brittle bone disease and dominant tumors. It also plans to apply the platform to other conditions through joint development and outlicensing deals.
The company still is keeping the details of its platform and its therapeutic strategy under wraps. ¿We¿re very conscious that we¿re very small, and we need to get a lead time,¿ Farrar said. However, its approach is based on combining gene therapy with ribozyme-based pharmaceuticals in order to suppress the pathological effects of dominant mutations.
The strength of Optigen¿s method lies in its potential to suppress multiple dominant mutations found in a particular allele in a non-site-specific manner. More than 150 different mutations in the rhodopsin gene can give rise to a dominant form of RP, for example, and developing individual therapies for each one is not feasible. Optigen also will look at ways of retarding apoptotic processes that accelerate the onset of RP.
The company does not yet have any people on the payroll, although an initial team of seven employees has been selected. The company is still negotiating terms with Trinity College Dublin. This process is expected to be completed early in the new year, Farrar said. Optigen then plans to raise seed funding of around EUR1.25 million, before seeking a more substantial round 12 months later. At that point, it also will look for a permanent CEO.