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UniQure Fabry Disease Gene Therapy Gets FDA IND Clearance

NEW YORK – Amsterdam-based UniQure announced on Wednesday that the US Food and Drug Administration cleared its investigational new drug application for AMT-191, a candidate gene therapy for Fabry disease.

Fabry disease is a rare lysosomal storage disorder caused by mutations in the GLA gene that result in the toxic accumulation of fatty acids in the liver through reduced or absent alpha-galactosidase enzyme activity. AMT-191 delivers an alpha-galactosidase A transgene directly to the liver via an adeno-associated virus type 5 vector.

UniQure plans to conduct a first-in-human Phase I/II multicenter, open-label clinical trial of the gene therapy candidate in the US. The trial will assess the therapy's safety, tolerability, and efficacy in two dose-escalation cohorts of three participants each.

"The clearance of the IND for AMT-191 represents a key milestone for the company, with four programs now in clinical phase," Walid Abi-Saab, chief medical officer of UniQure, said in a statement.

The company is currently developing gene therapies for amyotrophic lateral sclerosis and autosomal-dominant Alzheimer's disease, and is planning a Phase I study of a gene therapy for refractory mesial temporal lobe epilepsy, following FDA clearance for that IND last month.

Earlier this year, UniQure announced plans to press forward with late-stage development plans for a Huntington's disease gene therapy, despite inconsistent biomarker data reported in interim results from a Phase I/II trial that spooked investors and sent the company's stock tumbling.

Nonetheless, the company announced plans to cut over half of its research and technology projects, along with approximately 20 percent of its workforce, as part of a strategic reorganization.