Spark Therapeutics, a Philadelphia-based late clinical-stage gene therapy company, has entered into a license agreement with Selecta Biosciences, a Watertown, Massachusetts-based clinical-stage biopharmaceutical company, for the exclusive worldwide rights to Selecta’s proprietary Synthetic Vaccine Particles (SVP) platform technology. The SVP technology will be used in conjunction with gene-therapy targets. Gene-therapy targets include the factor VIII blood-clotting protein for hemophilia A as well as exclusive options for up to four additional undisclosed genetic targets.

The agreement is worth up to $460 million, under which 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 also 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.

The terms of this agreement do not apply to Spark Therapeutics’ ongoing investigational development programs in inherited retinal diseases (IRDs), including voretigene neparvovec, a compound for treating retinal pigment epithelium 65-mediated IRD, and SPK-7001, a compound for treating choroideremia. This agreement does not impact Spark Therapeutics’ ongoing Phase I/II trial of SPK-9001 in hemophilia B in collaboration with Pfizer or its planned Phase I/II trial of SPK-8011 in hemophilia A.

Selecta’s immune tolerance SVP 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, according to Selecta. Neutralizing antibodies form in response to an initial administration of an AAV gene therapy and are thought to prevent subsequent usage of the therapy, according to Selecta.

Source: Spark Therapeutics