Genetic medicines company Passage Bio, Inc. (PASG) announced Wednesday that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for PBML04, an adeno-associated virus (AAV)-delivery gene therapy that is being studied for the treatment of Metachromatic Leukodystrophy (MLD).
The clearance of this IND is based on preclinical data supporting PBML04 through Passage Bio's partnership with the University of Pennsylvania's Gene Therapy Program and the strong CMC and analytics capabilities developed together internally.
This IND clearance marks Passage Bio's fourth IND clearance as a company and its third pediatric lysosomal storage disorder program to reach clinical development.
The Phase 1 clinical trial will utilize intra-cisterna magna (ICM) administration to deliver an AAVhu68 capsid to express ARSA and potentially address both central nervous system and peripheral manifestations of this devastating disease.
MLD is a rare, fatal, pediatric, lysosomal storage disease that currently has limited available treatment options. MLD is caused by mutations in the arylsulfatase-A (ARSA) gene which reduces enzyme activity, leading to progressive build-up of toxic sulfatides in the central and peripheral nervous system. The estimated worldwide incidence of MLD is approximately 1 in 100,000 live births.
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