Companies Collaborate to Advance Dominant RP Gene Therapy

April 01, 2014

Two emerging gene-therapy development companies — Philadelphia-based Spark Therapeutics and Ireland-based Genable Technologies — have entered into a strategic partnership to accelerate the advancement of a gene therapy for people with autosomal dominant retinitis pigmentosa (adRP) caused by mutations in the gene RHO. The collaboration has the potential to provide additional product-development benefits to both companies in the future.

Under terms of the agreement, Genable will license Spark’s adeno-associated virus (AAV) gene-delivery system, which enables therapeutic genes to penetrate the patient’s retinal cells. The AAV will be used in Genable’s emerging gene therapy, known as GT038. Genable will also receive product development advice and expertise from Spark.

In turn, Spark will receive manufacturing revenues, milestone payments and royalties on future sales of GT038. Genable will also provide Spark with expertise in gene suppression strategies, which would benefit some of its early-stage product development programs for dominant diseases. Genable has developed gene-suppression technologies that shut down the production of harmful proteins which are characteristic of adRP and many other dominantly inherited conditions. 

“Our experience with AAV clinical trials and manufacturing can accelerate Genable’s efforts as well as the gene-therapy projects of other companies and academic groups,” says Jeffrey D. Marrazzo, co-founder, president and chief executive officer of Spark Therapeutics. “Our ultimate goal is to help as many patients as we can, so we are open to considering partnerships and agreements where our expertise can be helpful.”

“Likewise,” Marrazzo adds, “Genable’s gene-suppression technology is very attractive to us, as we are pursuing similar strategies to address dominant genetic diseases. Spun off last year from the Children’s Hospital of Philadelphia, Spark is conducting a Phase III clinical trial of a gene therapy for children and adults with Leber congenital amaurosis (LCA) caused by mutations in the gene RPE65. In the Phase I/II human study, the emerging therapy restored significant vision in participants who were virtually blind. Spark is developing gene therapies for a variety of conditions, including other inherited retinal diseases.

Established a decade ago, Genable is developing gene therapies for autosomal dominant diseases. GT038 is the company’s most advanced product, and has received orphan designations to bolster product marketing and clinical development in the United States and Europe.

“This collaboration is great news for people with retinal diseases, because it helps both companies move treatments into the marketplace,” says Stephen Rose, Ph.D., chief research officer of the Foundation Fighting Blindness. The Foundation provided significant funding for the development of LCA gene therapies and GT038. “It’s a win-win-win,” Dr. Rose adds.” Spark wins. Genable wins. And most important, the patients win.”