Gene therapy offers the potential for a long-lasting, if not permanent, treatment for an inherited disease, but cells that divide rapidly, such as those in the liver, present a thorny problem. Because of how they insert themselves in the cells, some forms of gene therapy get “diluted” as the cells divide.
It’s a particular problem in growing children. Cambridge, MA-based LogicBio says it has developed a workaround by combining gene editing with gene therapy. The firm has raised $45 million in additional capital to help bring this technology into human testing, and it is moving from California to the LabCentral shared incubator space in Cambridge’s Kendall Square.
LogicBio calls its technology GeneRide. The company says its approach can transfer genetic material to specific sites to repair a faulty genetic sequence. The company’s focus is metabolic disorders that affect the liver in children. Published research shows that metabolic disorders of the liver can progress to injury affecting other organs. In rare cases, the severity of the disease requires a pediatric liver transplant.
If GeneRide works as the company envisions, the gene therapy would offer a one-time treatment that avoids side effects.
London-based Arix Bioscience (LSE: ARIX) led the Series B round of investment, which was joined by new investors OrbiMed, Edmond De Rothschild Investment Partners, Pontifax, and SBI Japan-Israel Innovation Fund. Earlier investor OrbiMed Israel Partners also joined in the latest investment. In total, LogicBio says it has raised approximately $50 million in financing to date.
Gene therapy remains largely experimental. UniQure (NASDAQ: QURE) received the Western world’s first gene therapy approval in 2012 for alipogene tiparovec (Glybera), a treatment for a rare metabolic disorder. But earlier this year, the company, split between the Netherlands and Lexington, MA, announced it would not seek renewal of its conditional approval, set to expire in October. Patient demand for the drug was limited and the company did not expect that to change.
The first U.S. approval could come soon. Philadelphia-based Spark Therapeutics (NASDAQ: ONCE) is awaiting an FDA decision on a gene therapy for an inherited form of blindness. Cambridge-based Bluebird Bio (NASDAQ: BLUE) last week released early data from a Phase 3 study in patients with beta-thalassemia, a rare blood disorder.
The technologies underlying LogicBio’s approach were developed at Stanford University by company co-founders Mark Kay, Adi Barzel, and Leszek Lisowski. In addition to its Cambridge site, the company also has scientists in Tel Aviv, Israel.