MeiraGTx wins RPD designation for Leber candidate

23 January 2025

US genetic meds company MeiraGTx Holdings (Nasdaq: MGTX) yesterday revealed that the US Food and Drug Administration (FDA) has granted the company Rare Pediatric Disease (RPD) designation to its AAV8-RK-RetGC program for the treatment of patients with Leber congenital amaurosis due to GUCY2D mutations (LCA1).

Despite what appears to be a positive development for the company, MeiraaGTx, shres closed down more than 5% at $6.08.

This is the fourth Rare Pediatric Disease Designation that MeiraGTx has received in the last three months, including AAV8-RK-AIPL1 for the treatment of LCA4 retinal dystrophy, AAV8-RK-BBS10 for the treatment of Bardet-Biedl syndrome (BBS) due to BBS10 mutations and AAV5-RDH12 for the treatment of RDH12 associated retinal dystrophy.

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