March 7, 2016 8:26am

A Dublin, Ireland based company for $6 M in cash and 265 K ONCE shares 


 

ONCE has acquired Dublin, Ireland-based Genable Technologies, a private gene therapy innovator with which ONCE has collaborated since 2014 in the development of Genable's therapeutic program targeting one of the prevalent forms of inherited retinal disease.

 

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

·         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.

 

The Bottom Line: Payment Genable shareholders consisted of $6 M in cash and 265,000 shares of ONCE common stock. Additional financial terms were not disclosed.

Genable is developing gene-based therapies for the treatment of dominantly inherited genetic disorders. This class of incurable disorders comprises hundreds of individual members including different forms of autosomal dominant retinitis pigmentosa (adRP).

Dominant inheritance means that on average 50% of the children of an affected person will be affected by the disorder. Another common feature of these disorders is that the causative genes are often characterized by mutational heterogeneity. Therefore typically a large number of individual mutations in a single gene can lead to a particular disorder. For example any one of approximately 200 different mutations in the rhodopsin gene (RHO) can lead to adRP. Developing individual therapies for so many RHO mutations is practically impossible.

ONCE closed at $34.53 which was UP on Friday +$0.35