| 05 October 2015

Spark Therapeutics treatment for Form of LCA on course to become the first gene therapy to be approved in the USA

Spark Therapeutics have developed a potential gene therapy for a rare form of blindness known as Leber congenital amaurosis (LCA). This therapy is for a particular form of LCA known as LCA2, as it is caused by mutations in the RPE65 gene. Individuals with this condition experience night blindness as one of the earliest symptoms and this can deteriorate to severe vision impairment. There is currently no approved therapy LCA. Spark’s therapy, known as SPK-RPE65, replaces the defective gene by using viruses to deliver a working copy of the RPE65 gene directly to the patient’s eye.
In a study of 31 people with LCA2, 21 received the therapy, and 10 people were in the control group. After treatment, two thirds of individuals in the therapy group were able to successfully navigate a specially designed mobility course under very low light conditions indicating a restoration in their vision. There were no serious events during the trial related to the therapy and it is safe and well tolerated. The trial also showed that participants who received the treatment also outperformed those in the control group on two secondary goals: improving light sensitivity of the eye and change in mobility test score for the first eye treated. The third endpoint, which is the ability to read further on the standard eye chart didn’t result in a statistically significant result.
Only individuals with this form of LCA will benefit from this therapy as it is replaces the defective RPE65 gene, responsible for subtype 2 of the disease..
‘Spark will now look to engage with the US drug regulators, the FDA, in order to file for approval of the therapy next year. Retina International will report on developments as they arise to ensure our membership is kept fully informed’ said Christina Fasser, President of Retina International.

Join Our Mailing List