US biotech Spark Therapeutics has set up a UK office as it targets a rapid launch of its under-review gene therapy for a form of inherited blindness.
The Nasdaq-listed firm, which has a market cap of $2.4bn (£1.8bn), is at the forefront of the emerging global field of gene therapies for serious and rare diseases.
Their blindness treatment has found success in clinical trials, improving the sight of children with a rare disease known as Leber congenital amaurosis whose vision would otherwise have worsened or been lost over time. There is currently no available treatment for the condition.
Scientists have identified 200 genes where mutations lead to genetic blindness, affecting hundreds of thousands of people worldwide. It is hoped Spark’s gene therapy approach can ultimately be applied to find cures for almost all of these diseases.
The condition Spark’s treatment addresses arises from the mutation of just one of the 200 genes, known as RPE65. It affects up to 6,000 people worldwide.
The treatment works by injecting a normal functioning copy of the affected gene into the back of both of the patient’s eyes.
In clinical trials children who were unable to navigate an obstacle course in low light made big improvements just a year later after receiving the therapy.
You hear the children talk about being able to do things you and I don’t have to think about on a daily basis. It’s incredibly gratifying
Jeff Marazzo, Spark
Spark’s treatment has been submitted for approval with US and EU drug regulators. A decision is expected in the US first, where it would be the country’s first available gene therapy.
In Europe it is set to be the third to launch, and the first to address inherited blindness. A verdict is expected in the US next January and in the EU later in the year.
Jeff Marazzo, Spark chief executive, told The Daily Telegraph the firm was engaged in “pre-launch activity in Europe” and had hired a commercial and medical diagnostic team based in Paddington, west London.
Commenting on the firm’s clinical successes to date, Mr Marazzo said: “You hear the children talk about being able to do things you and I don’t have to think about on a daily basis. It’s incredibly gratifying”.
The company is already trialing a gene therapy for a second type of inherited blindness, where men in their second or third decade of life experience a deteriorating field of vision reducing down to a pinhole.