GENABLE TECHNOLOGIES LTD, announces that it has been acquired by Spark Therapeutics, Inc.

Dublin, 7th March 2016 – Genable, an Irish bio-pharmaceutical company developing novel gene therapies for the treatment of dominant genetic diseases, today announced that it has completed a transaction which will see it become fully owned by Spark Therapeutics, a world leader in gene therapy development.

Annette Clancy, Chair of the Board of Directors of Genable commented "We are delighted that this transaction with Spark Therapeutics Inc. will ensure the expedient clinical development of RhoNova™ for the treatment of autosomal dominant rhodopsin linked retinitis pigmentosa (RHO-adRP), a leading cause of inherited blindness. The unanimous recommendation of the Board was that Spark Therapeutics, world leaders in AAV gene therapies, has the relevant resources and expertise to maximise the chance of success for RhoNova™ and deliver its benefit for patients. We wish them every success."

"The longstanding support of our patient, institutional and funding partners and scientific colleagues has been central to expediting development of RhoNova™ from its academic roots in Trinity College Dublin through to today. Spark Therapeutics, a global leader in AAV-based gene therapy, has collaborated with Genable since 2014 in the development of RhoNova™, our Product has and will continue to greatly benefit from Spark's knowledge and technology platform" said Professor Jane Farrar, co-founder and CSO of Genable.

Genable Technologies Ltd will become a wholly owned subsidiary of Spark Therapeutics Inc. and the Company will remain situated in Ireland.

Genable were supported in executing this transaction by its legal counsel Mason, Hayes and Curran (Ireland) with input from Choate, Hall & Stewart LLP (US). Stifel, Nicolaus & Company, Incorporated is acting as financial advisor to Genable.

Background: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.

About Genable Technologies

Genable Technologies Ltd is a privately held, venture-capital backed, Dublin (Ireland), based gene therapy company. The Company is developing new gene therapies to treat "dominant" genetic diseases using technologies initially generated in Trinity College Dublin. The Company has received significant support and investment from Fountain Healthcare Partners, Delta Partners, Fighting Blindness Ireland, Foundation Fighting Blindness Clinical Research Institute (U.S.), Trinity College Dublin and Enterprise Ireland. To learn more please visit

About Spark Therapeutics

Spark is a gene therapy leader seeking to transform the lives of patients with debilitating genetic diseases by developing one-time, life-altering treatments. Spark's initial focus is on treating rare diseases where no, or only palliative, therapies exist. Spark's most advanced product candidate, SPK-RPE65, which has received both breakthrough therapy and orphan product designation, recently reported positive top-line results from a pivotal Phase 3 clinical trial for the treatment of rare blinding conditions. Spark's validated gene therapy platform is being applied to a range of clinical and preclinical programs addressing serious genetic diseases, including inherited retinal dystrophies, hematologic disorders and neurodegenerative diseases. Spark builds on two decades of research, development and manufacturing at The Children's Hospital of Philadelphia, including human trials conducted across diverse therapeutic areas and routes of administration. To learn more, please visit