Spark Therapeutics recently dropped a press release with some exciting developments in gene therapy treatments. However for the uninitiated, the report can be a little dense, so let’s break it down…
The big takeaway is that Spark has announced that they have submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for a one-time gene therapy treatment for patients with the degenerative retinal disease Leber congenital amaurosis (LCA) or retinitis pigmentosa (RP). The trade name for this treatment will likely be LUXTURNA™.
On this side of the pond, the well known Food and Drug Administration (FDA) has the treatment under Priority Review, meaning we will hopefully be seeing it on the market sooner than later.
John Furey, the chief operating officer of Spark Therapeutics, stated, in regards to the near future, "For the first time, these individuals, who eventually will progress to complete blindness, have hope for a potential treatment option that may restore their vision.” These are exciting times.
This has been a long road, the first trials began in 2007, and before that many years of hard research and laboratory work were needed to get us to where we are today. The most recent trial took place between 2013 and 2015, involving 41 participants aged 4 to 44. For the most part, of those 41 participants, a majority showed significant improvement in sight when compared to the control group. Fortunately, no serious adverse effects were observed in participants who received the treatment. This is why Spark has been able to move forward with their research.
It is important to us that everyone understands the tangible effect of buying our shirts. Some of the early research for this therapy (since the early 1990s and up until the first clinical trial) was funded by the Foundation Fighting Blindness, an organization we work very closely with. So where does Two Blind Brothers fit in? Our goal is to find these researchers who have a great hypothesis, a little bit of a proof of concept and who are trying to get ready for a clinical trial. If we can find a healthcare venture fund or a biotech fund that can come in and fund all those expenses to get that therapy into a commercial space we have done our job by giving these projects wings to get going.
Without the generosity we see from all of you on a daily basis, things like this would not be possible. We are continually grateful to all of you. Let’s keep fighting together!