Date: 2016-12-05
Type of information: Licensing agreement
Compound: Synthetic Vaccine Particles (SVP™) platform technology
Company: Spark Therapeutics (USA - PA) Selecta Biosciences (USA - MA)
Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases
Type agreement: licensing
Action mechanism: gene therapy. 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.
Disease: hemophilia A and up to four additional undisclosed genetic targets
Details: * On December 5, 2016, Spark Therapeutics and Selecta Biosciences 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. The terms of this agreement do not apply to Spark Therapeutics' ongoing investigational development programs in inherited retinal diseases (IRDs), including voretigene neparvovec for the treatment of RPE65-mediated IRD and SPK-7001 for choroideremia. This agreement does not impact Spark Therapeutics' ongoing Phase 1/2 trial of SPK-9001 in hemophilia B in collaboration with Pfizer or its planned Phase 1/2 trial of SPK-8011 in hemophilia A. Selecta independently is applying its SVP technology to its own proprietary gene therapy programs. Selecta has obtained an exclusive license from Massachusetts Eye and Ear to Anc80, an in silico-designed gene therapy vector, for Methylmalonic Acidemia and has options for additional pre-defined indications. Additionally, Selecta is advancing a proprietary gene therapy program for Ornithine Transcarbamylase Deficiency.
Financial terms: 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.
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