Gene therapy safe, improves vision in X-linked retinitis pigmentosa in phase 1/2 study
Botaretigene sparoparvovec, an investigational gene therapy, was safe and improved vision in subjects with X-linked retinitis pigmentosa with disease-causing variants in the RPGR gene, according to a press release from MeiraGTx.
Top-line data from the MGT009 phase 1/2 clinical study found that botaretigene sparoparvovec, formerly known as AAV-RPGR, was generally safe and well tolerated. In addition, study participants experienced significant improvements in retinal function, visual function and functional vision at 6 months compared with a randomized untreated control arm.
“We are extremely encouraged by these data, which demonstrate improvement following treatment with botaretigene sparoparvovec compared to an untreated randomized control group in a range of endpoints that are relevant to this severe disease,” Alexandria Forbes, PhD, president and CEO of MeiraGTx, said in the release. “These data give us increased confidence in the potential for botaretigene sparoparvovec to meaningfully improve the lives of the thousands of patients with XLRP.”
Botaretigene sparoparvovec, in joint development by MeiraGTx and Janssen Pharmaceuticals, is under evaluation in the phase 3 Lumeos study, which is currently dosing subjects who have XLRP with disease-causing variants in the RPGR gene.
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