MeiraGTx Gene Therapy Shows Promise in Restoring Vision for Children with Severe Retinal Dystrophy

Eleven children who were born legally blind experienced significant improvements in visual acuity following the administration of MeiraGTx’s investigational gene therapy. This development pertains specifically to AIPL1-associated severe retinal dystrophy. Encouraged by these promising findings, the biotech firm is now seeking expedited regulatory approval.

On February 21, The Lancet published findings related to four of the treated children. According to the study, the treated eyes demonstrated noticeable improvements in visual acuity within a timeframe of four weeks or more. In contrast, the untreated eyes of these children continued to deteriorate, with their vision eventually becoming unmeasurable. The study further highlighted that the benefits of this therapy were not confined solely to visual enhancement. Researchers reported broader positive effects on various aspects of child development, including improved communication skills, behavioral adjustments, educational progress, emotional well-being, and increased social integration. Alexandria Forbes, Ph.D., CEO of MeiraGTx, underscored these additional benefits in the company’s statement accompanying the publication.
However, one child experienced cystoid macular edema, a condition characterized by the accumulation of fluid in the macula of the treated eye. While this complication was noted, researchers observed that it partially improved over time and did not hinder the child’s overall vision improvement. No further safety concerns were documented in the study.
Analysts from Evercore ISI described the results as strong clinical data that clearly demonstrate the therapy’s efficacy in addressing a significant unmet medical need in pediatric patients. In a report dated February 21, they noted that the treatment’s safety profile aligned with what is typically expected from a subretinal approach.

After engaging in discussions with the United Kingdom’s regulatory authorities, MeiraGTx has announced its intention to seek fast-tracked approval for the therapy in that country. Simultaneously, the company has been in dialogue with the U.S. Food and Drug Administration to explore pathways for accelerated approval in the United States.
The mechanism of the therapy involves the use of an adeno-associated virus to deliver functional copies of the AIPL1 gene directly into the eye via a single injection. This approach is designed to correct the underlying genetic deficiency responsible for retinal dystrophy.
The study began with an initial group of four children, aged between one and three years, who were all diagnosed with severe retinal dystrophy linked to mutations in the AIPL1 gene. Each of these children received treatment in one eye. Following the observation of positive safety outcomes and initial success, the trial was expanded to include an additional seven children, who subsequently received the therapy in both eyes.