November 5, 2015
By Alex Keown, BioSpace.com Breaking News Staff
PHILADELPHIA – The greater Boston area is getting a little bit more crowded as the gene therapy company is opening a satellite office in Waltham, Mass. to support the growth of several departments, including the commercial, medical, patient advocacy and business development groups.
The expansion into Boston comes less than one year following one of the largest biotech initial public offerings of $161 million and only a few weeks following a successful Phase III trial for its lead program, SPK-RPE65, for the treatment of RPE65-mediated inherited retinal dystrophies. The company plans to submit a Biologics Licensing Application to the U.S. Food and Drug Administration (FDA) in 2016. SPK-RPE65 has orphan designation in the United States (U.S.) and the European Union, and breakthrough therapy designation in the U.S.
SPK-RPE65 has the potential to address IRDs caused by mutations in the RPE65 gene, for which there currently are no pharmacologic treatments, Spark said on its website. Mutations in the RPE65 gene are linked to subtypes of Leber’s congenital amaurosis (LCA type 2) and retinitis pigmentosa (RP type 20). The company said it estimates there are approximately 3,500 individuals in the United States and five major European markets with these forms of IRDs.
“Over the past year, Spark has doubled in size, and we are continuing to expand to meet the needs of our growing business,” Jeffrey Marrazzo, co-founder and chief executive officer of Spark Therapeutics , said in a statement. “With successful completion of the Phase III pivotal trial of our lead gene therapy product candidate last month, we are building out teams in both offices to support commercialization activities and the future expansion of our growing pipeline. Our presence in the greater Boston area puts Spark in a prime position to readily access top talent in both burgeoning biotech hubs.”
Since its IPO earlier this year, Spark’s has grown by about 20 percent, hitting a high of $79.50 per share. It’s currently trading at $56.55 per share.
With the expansion into the Boston-area, the company will look to hire some additional staff to support the satellite office. Initially the office will have six employees, but will hire “people in commercial, medical, patient advocacy and business development,” the Boston Business Journal reported this morning.
As Spark moves into Boston, it will have some work to do to elbow aside competitors in gene therapy, including bluebird bio , which specializes in treatments for blood disorders and immuno-oncology through the use of gene therapy. The 23-year-old bluebird has a number of therapeutics in its pipeline. In June, bluebird struck a deal with Kite Pharma, Inc. to develop T-cell therapies to treat cancer linked to Human papilloma virus (HPV). T-cell receptors are showing promise in boosting the body’s own immune system to combat cancer. In other treatment areas, bluebird released a report in the spring showing its sickle cell anemia drug, LentiGlobin, has kept two patients transfusion free for 14 and 11 months, respectively. In June, LentiGlobin was also shown to be effective on a French teen, who was able to avoid getting a transfusion for three months.
One reason Spark and other gene therapy companies have located to the Boston area is the support offered by MassBiologics and its Fall River facility, which is able to manufacture the viral vectors needed to deliver the genes, the Journal said.
