Eleven children born blind at birth have regained visual acuity after receiving MeiraGTx’s investigational gene therapy, prompting the biotech to seek accelerated approval for AIPL1-associated severe retinal dystrophy.
The results of four children were published in The Lancet on February 21, showing significant improvements in treated eyes after four weeks. Treated eye acuity improved, while untreated eye acuity became unmeasurable.
MeiraGTx’s CEO said the treatment had life-changing benefits beyond vision improvement, including communication, behavior, schooling, and social integration. However, one child developed cystoid macular edema, a build-up of fluid in their treated eye, which partially improved over time.
Analysts described the findings as “strong clinical data” with clear evidence of efficacy. The company plans to submit the gene therapy for expedited approval in the UK and is discussing accelerated approval with the FDA in the US.
Source: https://www.fiercebiotech.com/biotech/meiragtx-gene-therapy-improves-vision-eleven-children-company-eyes-accelerated-approval