|Spark Therapeutics CEO Jeffrey Marrazzo|
Philadelphia's Spark Therapeutics ($ONCE) picked up a Dublin-based gene therapy outfit for just over $15 million to bring the company's technology into its own portfolio and together develop a treatment for an orphan inherited retinal disease.
Genable Technologies specializes in its RhoNova technology, which is designed to treat rhodopsin-linked autosomal dominant retinitis. Its success depends on an approach based on both suppression and replacement, according to the company's website, so that it can use a single therapy to treat all of the mutations in a particular gene and at the same time correct the genetic defect.
In other words, it suppresses a section of mRNA using RNA interference and it replaces it with a new section of mRNA that encodes the normal protein. Cutting a section of the gene and immediately replacing it with a healthy version is a tricky delivery concept, but an important genetic strategy for certain diseases.
Spark, which provided $6 million up front and the rest in its own shares, has worked with Genable's technology since it partnered with the company in 2014.
"We are excited to apply our deep expertise in AAV clinical development and manufacturing to augment Genable's great work, and expand the number of debilitating diseases of the eye that can be addressed through gene therapy," said Spark CEO Jeffrey Marrazzo.
- here's the release