Science/Health

Spark Therapeutics Buys Genable Technologies, Seeks To Cure Inherited Retinal Disease

Spark Therapeutics announced on March 7 that it has acquired for $15.1 million, Dublin, Ireland-based Genable Technologies. Both companies work in the realm of gene based therapy.

No other financial terms were disclosed.

Spark was started with a $50 million capital commitment from The Children's Hospital of Philadelphia (CHOP).

Genable has been working on a therapeutic program on "one of the most prevalent forms of inherited retinal disease, rhodopsin-linked autosomal dominant retinitis pigmentosa, that routinely leads to visual impairment and in the most severe cases to blindness." Spark has been collaborating with Genable on this program since 2014.

At present about 12,000 people in the United States suffer from this condition which has no approved pharmacologic treatment.

"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," read a press release announcing the acquisition.

Genable's team, comprised of professors Jane Farrar and Peter Humphries and opthalmologist Dr. Paul Kenna have been researching retinis pigmentosa for more than 20 years.

"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," said Jeffrey D. Marrazzo, Spark co-founder and chief executive officer.

"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," said Dr. Katherine A. High, co-founder, president and chief scientific officer of Spark.

The deal would "ensure the expedient clinical development of [its drug] RhoNova," said Genable chairwoman Annette Clancy.

Rhodopsin-linked autosomal dominant retinitis pigmentosa is "one of the most prevalent forms of inherited retinal disease."

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