Spark Therapeutics (NASDAQ:ONCE) and Selecta Biosciences, Inc. (NASDAQ:SELB) today announced a license agreement that provides Spark Therapeutics with exclusive worldwide rights to Selecta’s proprietary Synthetic Vaccine Particles (SVP™) platform technology for co-administration with gene therapy targets, including FVIII for hemophilia A, as well as exclusive options for up to four additional undisclosed genetic targets.
Selecta’s immune tolerance SVP, including SVP-Rapamycin, is an investigational technology intended to suppress the formation of neutralizing antibodies to an adeno-associated virus (AAV) capsid when used in combination with gene therapies, without altering the therapeutic profile of the gene therapy. Neutralizing antibodies form in response to an initial administration of an AAV gene therapy and prevent effective subsequent usage. The potential ability to re-dose a gene therapy may be beneficial where a patient has not achieved a sufficient therapeutic expression of the transferred gene in the initial dose.
“Selecta’s nanoparticle technology, which is undergoing preclinical testing in gene therapy, may prevent formation of neutralizing antibodies, and thus potentially enable re-dosing up to an optimal therapeutic profile by extending the reach of gene therapy to diseases that require higher doses or more extensive transduction of target cells than may be achieved through one-time dosing,” said Jeffrey D. Marrazzo, chief executive officer of Spark Therapeutics. “Importantly, if proven successful, the co-administration of Selecta’s technology with a gene therapy may enable repeat dosing of AAV gene therapies in both adults and pediatric patients, potentially minimizing the risk of a T-cell immune response to the capsid.”
“Gene therapy is a core area of focus for Selecta; one that we believe could benefit profoundly from our immune tolerance SVP technology platform,” said Werner Cautreels, Ph.D., president, CEO and chairman of Selecta. “We are excited about this license agreement with Spark Therapeutics, a recognized gene therapy leader, which accelerates the application of our SVP platform in gene therapy. Our preclinical studies in this field, together with the clinical data we have generated with SEL-212 in gout showing prevention of anti-drug antibodies, suggest that the application of our immune tolerance SVP technology to biologic therapies may greatly benefit patients with life-threatening diseases who currently lack adequate treatment options due to the occurrence of undesired immune responses.”
Subject to the terms of the agreement, Spark Therapeutics will make an initial $10 million cash payment to Selecta and purchase $5 million of Selecta’s common stock. Within 12 months of the agreement’s signing, Spark Therapeutics has agreed to pay Selecta an additional $5 million in cash and to purchase $10 million of Selecta’s common stock. Selecta will be eligible for up to $430 million in milestone payments for each target, with up to $65 million being based on Spark Therapeutics’ achievement of specified development and regulatory milestones and up to $365 million for specified commercial milestones. In addition, Spark Therapeutics will pay Selecta tiered mid-single to low-double-digit royalties on worldwide annual net sales of any resulting commercialized gene therapy.