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Spark Therapeutics announces plans to have 10 gene therapy clinical programs in place by 2018, including up to three ophthalmic indications

Spark Therapeutics (NASDAQ:ONCE), a gene therapy company based in Philadelphia, USA, has announced a three year vision to expand the company’s gene therapy portfolio and launch its first commercial product in 2017. The company’s lead gene therapy product, termed “SPK-RPE65”, is a replacement gene therapy treatment for RPE65-mediated inherited retinal degeneration. Spark had previously announced that their SPK-RPE65 pivotal trial, conducted in 31 patients, met its primary endpoint (p = 0.001), indicating an improvement of functional vision in the treated patients, compared to control subjects.


Announcing the goals at JP Morgan’s 34th Annual Healthcare Conference in San Francisco, Spark will aim to have 10 clinical programs in progress by 2018, including the introduction of two new programmes targetting Leber’s hereditary optic neuropathy (LHON) and Huntington’s disease (HD). Commenting on the clinical objectives, Chief Executive Officer, Jeffrey D. Marrazzo stated that results to date “reflect the power of a true platform that combines proven capabilities across vector selection, design and manufacture, a history of collaborating with regulators to optimize clinical development and develop novel clinical endpoints, and our position at the forefront of shaping a patient-centric, commercial model for gene therapies.”


At AAO’s 2015 meeting in Las Vegas, results from the company’s Leber’s congenital amaurosis (LCA) study showed that patients treated with replacement RPE-65 gene therapy were capable of navigating an obstacle course under varying light conditions. According to secondary endpoint data, treated subjects achieved a mean improvement of approximately two lines (9.0 letters averaged across both eyes) on the logarithm of the minimum angle of resolution (logMAR) scale, compared with a “slight improvement” (1.6 letters) seen in control subjects. Approximately 30% of the treated patients (numbering seven of twenty) experienced a 15-letter, or three-line, improvement in the first eye administered, compared with no improvement in the control group. In the second eye administered, four of twenty treated patients achieved a 15-letter improvement, in comparison to no improvement seen in the control subjects.


Programmes highlighted by the company included:

  • SPK-RPE65: for the treatment of Leber’s congenital amaurosis;
  • SPK-CHM: for the treatment of choroideremia;
  • SPK-FIX: for the treatment of hemophilia B;
  • SPK-FVIII: for the treatment of hemophilia A; and
  • SPK-TPP1: for the treatment of TPP1 deficiency, a form of Batten disease.