Trinity’s Gene Medicine Spin-Off Genable Technologies Sold to Spark Therapeutics

Genable Technologies, an Irish bio-pharma company developed at Trinity College Dublin in 2003, which specialises in developing gene medicines to target genetic diseases, has been sold to US gene therapy leader, Spark Therapeutics.

Genable Technologies has collaborated with Spark since 2014 in the development of a therapeutic program that targets one of the most prevalent forms of inherited retinal disease (IRD).

With the acquisition, Spark acquires RhoNova™, a potential treatment targeting rhodopsin-linked autosomal dominant retinitis pigmentosa (RHO-adRP), an IRD that routinely leads to visual impairment and in the most severe cases to blindness.

RhoNova™ is designed to both suppress the expression of a faulty gene and deliver normal copies of the RHO gene to restore normal expression. RhoNova™ has been granted Orphan Drug Designation in both the US 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 12,000 patients in the United States and the five major European markets (EU5).

Professor of Genetics at Trinity, Jane Farrar, has been instrumental in the creation and development of Genable Technologies over the past 13 years.

Professor Farrar said: “The journey was started by Irish RP patients many years ago, was navigated by a great team of scientists in Trinity and then developed and shaped by Genable Technologies. Without doubt at this juncture Spark Therapeutics provides the optimal way for this innovative therapy to progress to patients”.

Director of Trinity Research & Innovation, Diarmuid O’Brien, added: “It is wonderful to see Trinity research translating to enable impact for patients."

"It is important to recognise that the research of Jane Farrar, Pete Humphries and Paul Kenna in ocular genetics in Trinity has been supported by multiple sources including Science Foundation Ireland, the Health Research Board, Fighting Blindness Ireland, Foundation Fighting Blindness (USA), Enterprise Ireland, the Wellcome Trust, the European Research Council (ERC) and EU framework programmes. These funding streams have all enabled this success.”

Spark co-founder and chief executive officer, Jeffrey D. Marrazzo, said: “This transaction advances our strategy of leveraging Spark’s best-in-class gene therapy platform through a combination of internal innovation, acquisition and collaboration. Genable’s technology and promising RhoNova™ development program further strengthens our portfolio of treatments for IRDs, which is led by our Phase 3 program for RPE65-mediated blindness which recently reported overwhelmingly positive pivotal stage data.”

Shareholders in Genable Technologies received $6 million following the sale, along with 265,000 shares of Spark common stock. Additional financial terms were not disclosed.

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Thomas Deane, Media Relations Officer | deaneth@tcd.ie | +353 1 896 4685