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Study of SPK-RPE65 gene therapy shows gains in visual acuity
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LAS VEGAS — SPK-RPE65 treatment may result in rapid postoperative recovery of visual acuity for RPE65-mediated inherited retinal dystrophies, according to a presentation here.
Albert M. Maguire, MD, discussed the phase 3 study’s primary and secondary endpoints, and presented additional results for the gene therapy at the American Academy of Ophthalmology annual meeting.
As previously reported, the phase 3 clinical trial or SPK-RPE365 (Spark Therapeutics) showed a statistically significant improvement in the gene therapy intervention group compared with the control group in the primary endpoint — change in bilateral mobility testing between baseline and 1 year — and the first two secondary endpoints — full-field light sensitivity threshold testing and mobility testing for the assigned first eye.
As a third secondary endpoint, the treatment group achieved a mean improvement of approximately 9 letters, compared with 1.6 letters of improvement in the control subjects.
Seven of 20 patients improved 15 letters in the first eye of the treatment group compared with none in the control group, and 4 of 20 treated patients reached the same improvement in the second eye, compared with none in the control group. — by Kristie L. Kahl
Reference:
Maguire AM. Phase 3 trial of AAV2-hRPE65v2 (SPK-RPE65) to treat RPE65 mutation-associated inherited retinal dystrophies. Presented at: American Academy of Ophthalmology annual meeting. Nov. 14, 2015; Las Vegas.
Disclosure: Maguire reports he is a consultant for Novartis Pharmaceuticals.