One of the most promising applications of gene therapy is in disorders of the eye – which informs a recent acquisition by Philadelphia-based Spark Therapeutics.
Though it’s gotten press for its gene therapy approach to treating hemophilia, Spark Therapeutics just bought out Irish gene therapy outfit Genable Technologies – with whom it’s been collaborating since 2014 – for about $15 million in stock.
The deal involves Genable receiving some $6 million in cash and 265,000 shares of Spark (NASDAQ: ONCE) stock.
Spark now has control of RhoNova, a therapy for autosomal dominant retinitis pigmentosa – a degenerative vision disorder that can often lead to blindness. The Genable drug has received orphan drug status in both the U.S. and Europe, using an adeno-associated viral vector that was developed by Spark.
There are no approved therapies for this form of retinitis pigmentosa, which treats about 12,000 patients in the U.S. and five European markets, Spark says.
Spark’s portfolio centers around correcting genetic diseases of the eye, blood and central nervous system. Its most advanced trial is in Phase 3, treating RPE65-mediated inherited retinal dystrophies. It’s also pursuing therapies of hemophilia and Batten disease, a rare lysosomal storage disorder.
