Genable Technologies Acquired by Spark Therapeutics

Patient Led Research Moves Closer to the Clinic

Fighting Blindness is very pleased with the announcement that Spark Therapeutics, a US based gene therapy company, have acquired Genable Technologies Ltd, a spin-out campus company from Trinity College Dublin of which the charity were early supporters. Kevin Whelan, Chief Executive of Fighting Blindness commented on the acquisition saying: “Fighting Blindness has funded basic genetic research into inherited retinal degenerations for over 30 years, and we are thankful to the early leaders of the charity who saw the potential in the outstanding research in Trinity College Dublin and took the opportunity to be involved with Genable Technologies. We are very excited to see the work reach this stage of therapy development and would like to congratulate all those involved in Genable on this success.”

Commenting further, Kevin Whelan stated “The goal of Fighting Blindness, a patient-led organisation, is to find treatments and cures for conditions causing vision loss. This announcement moves RhoNova™, a potential treatment targeting one of the most prevalent types of retinitis pigmentosa, further towards clinical application and closer to the people who will benefit from it. Any financial benefit accruing to Fighting Blindness from this sale will be committed to advancing our research mission. This is a prime example of the power of patient involvement and how patient-led research can make a real impact in the development of therapies. We are very proud in Fighting Blindness, and on behalf of our members, to have played a part in that.”

Fighting Blindness will keep members and supporters fully informed of any further developments as it continues to work hard to fulfil its goals to cure blindness, support people living with sight loss, and empower patients.

Background

Retinitis pigmentosa (RP) refers to a group of inherited diseases that affect the photoreceptor (light sensing) cells responsible for capturing images from the visual field. In Ireland, it is estimated that one in 4,000 people has RP, which equates to approximately 1,500 individuals. RP is one of the most complicated genetic conditions of all, and over 50 different genes have been identified to be causative for various forms of RP. The type of RP which Genable has been developing a therapy for is rhodopsin-linked autosomal dominant retinitis pigmentosa (RHO-adRP). The team behind Genable – Prof Jane Farrar, Prof Peter Humphries and ophthalmologist Dr Paul Kenna and the dedicated Ocular Genetics Unit research group – have spent over 20 years investigating this form of retinitis pigmentosa (RP).

RhoNova™ is a treatment developed by Genable to target rhodopsin-linked autosomal dominant retinitis pigmentosa (RHO-adRP), an inherited retinal disease (IRD) that leads to blindness in many cases. Using adeno-associated virus (AAV) vectors developed and manufactured at Spark, RhoNova™ is designed to suppress expression of the faulty gene and deliver the normal rhodopsin (RHO) gene thereby providing a therapy that is independent of the specific causative rhodopsin mutation. RhoNova™ has been granted Orphan Drug Designation in both the U.S. and Europe, in addition to the Advanced Therapy Medicinal Product designation from the European Medicines Agency. There is currently no approved pharmacologic treatment for RHO-adRP, which affects an estimated 30,000 patients worldwide.

Fighting Blindness

March 11, 2015