Patient led research moves closer to the clinic

Fighting Blindness welcomes the announcement that Spark Therapeutics, a US based gene therapy company, has acquired Genable Technologies Ltd. Fighting Blindness has funded basic genetic research into inherited retinal degenerations for over 30 years and were early supporters of Genable, which is a spin-out campus company from Trinity College Dublin (TCD).

We are thankful to the early leaders of Fighting Blindness who saw the potential in the outstanding research in TCD and took the opportunity to be involved with Genable. It is very exciting to see that work reach this stage of therapy development and we congratulate all those involved in Genable on this success.

Genable Technologies Ltd will become a wholly owned subsidiary of Spark Therapeutics Inc. and the company will remain situated in Ireland. With the acquisition, Spark acquires Genable’s lead therapy, RhoNova™, a potential treatment targeting one of the most prevalent types of retinitis pigmentosa (RP).

The team behind Genable includes Prof Jane Farrar, Prof Peter Humphries and Dr Paul Kenna (pictured right), who have spent over 20 years investigating genetic eye diseases, including RP. 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 the condition. The type of RP which Genable has been developing a therapy for is rhodopsin-linked autosomal dominant retinitis pigmentosa (RHO-adRP). The name of this potential therapy is RhoNova™.

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.

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.

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