Opus Genetics Inc. has secured U.S. FDA alignment on an eight-patient phase III trial of its lead gene therapy, OPGx-LCA5, for an ultra-rare form of inherited childhood blindness.
The gene therapy specialist formerly known as EG427 has a new name and fresh momentum, after closing a €33 million (US$37.7 million) series C that will further advance clinical development of the lead program in chronic neuro-urology indications.
Metachromatic leukodystrophy (MLD) is a rare inherited lysosomal storage disorder characterized by progressive neurodegeneration resulting from loss of arylsulfatase A (ARSA) activity. Researchers at Kazan Federal University reported preclinical efficacy data for a gene therapy candidate in a porcine model of MLD.
