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