MeiraGTx Holdings Plc. (MGTX), a clinical-stage genetic medicines company, on Friday announced the progress in its gene therapy program for Leber Congenital Amaurosis 4 or LCA4, a severe retinal dystrophy caused by a genetic deficiency of AIPL1.
The Phase I/II trial data, recently published in the Lancet, highlights the exceptional efficacy of rAAV8.hRKp.AIPL1, a one-time gene therapy that has shown substantial improvements in visual acuity and functional vision in young children, previously legally blind at birth.
The therapy, delivered through a sub-retinal injection, has demonstrated significant protection against retinal degeneration, with all 11 children treated showing improved vision after just one administration.
MeiraGTx said this data marks a crucial milestone in the company's commitment to transforming treatment options for inherited retinal diseases or IRDs.
The company is currently working with global regulators, including the MHRA and FDA, to accelerate the approval process and provide rAAV8.hRKp.AIPL1 to children born with LCA4.
"LCA4 is one of the most severe forms of inherited blindness, and the results are truly remarkable. These improvements go beyond vision, offering life-changing benefits in communication, behavior, schooling, and social integration.
MeiraGTx said it is on track to advance its innovative genetic medicines and remains focused on bringing life-changing therapies to patients with high unmet needs in the field of ocular genetics.
MGTX closed Thursday's (Feb.20 2025) trading at $6.83 down by 0.87%. In premarket trading Friday the stock is down by 3.37% at $6.60.
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