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Spark Therapeutics buys Genable Tech for $6 million in cash and stock deal

pharmafile | March 11, 2016 | News story | Business Services, Medical Communications Genable Technologies, Spark Therapeutics, cash & stock, genetic technologies, merger & acquisitions 

Spark Therapeutics (Nasdaq: ONCE) said it has acquired Ireland-based Genable Technologies, a privately-held gene therapy firm for $6 million in a cash and stock deal.

Genable shareholders will also receive 265,000 shares in Spark, the company said in a statement. No additional financial terms were disclosed.

Spark Therapeutics has collaborated with Genable since 2014 to develop Genable’s therapeutic program targeting one of the most prevalent forms of inherited retinal disease (IRD).

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Jeffrey Marrazzo, Spark co-founder and chief executive, 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 III program for RPE65-mediated blindness which recently reported overwhelmingly positive pivotal stage data.”

The deal gives Spark access to RhoNova, a potential gene-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.

Using an adeno-associated virus (AAV) vector developed and manufactured at Spark, RhoNova is designed to both suppress the expression of a faulty gene and deliver normal copies of the RHO gene to restore normal expression.

Katherine High, co-founder, president and chief scientific officer at Spark, said: “Genable has provided convincing data in animals to support their innovative scientific approach for the treatment of autosomal dominant diseases, where it is critical to both suppress faulty genes and replace them with a functional copy in order to have a meaningful therapeutic effect. We look forward to using our validated gene therapy development platform and expertise to accelerate the clinical development of RHoNova, while examining other potential applications for the Genable suppression/ augmentation approach outside of this initial indication.”

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).

Anjali Shukla

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