Gene-Edited Retinal Regeneration Therapy RetinaGen Receives FDA Breakthrough Therapy Designation: Photoreceptor In Situ Regeneration Achieved for the First Time
Editas Medicine's RetinaGen gene-editing therapy received FDA Breakthrough Therapy Designation, achieving in situ regeneration of retinal photoreceptors in humans for the first time, offering hope of restored vision for patients with inherited blindness.
Gene-Edited Retinal Regeneration Therapy RetinaGen Receives FDA Breakthrough Therapy Designation: Photoreceptor In Situ Regeneration Achieved for the First Time
Editas Medicine announced on July 10 that its RetinaGen gene-editing therapy received FDA Breakthrough Therapy Designation. The therapy uses an AAV vector to deliver the CRISPR-Cas9 system to the retina, reactivating dormant Muller glial cells and reprogramming them into photoreceptors — achieving in situ regeneration of retinal photoreceptors in humans for the first time.
In a Phase I/II clinical trial for patients with Leber congenital amaurosis, 9 of 12 treated patients showed measurable vision improvement within 6 months of injection, with 3 patients recovering from light perception only to facial expression recognition levels. This marks the first time gene editing has achieved functional regeneration of lost photoreceptors in humans.
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