PASG - Passage Bio on go with PBGM01 gene therapy trial in the U.S.

The FDA has signed off investigational new drug ((IND)) application for Passage Bio's (PASG) lead product candidate, PBGM01, an adeno-associated virus-delivery gene therapy that is being studied for the treatment of infantile GM1 gangliosidosisDosing in Imagine-1 Phase 1/2 study is expected to commence in 1Q 2021.The trial is a global open-label, dose escalation study of PBGM01 administered by a single injection into the cisterna magna in pediatric subjects with early and late infantile GM1. The clinical program will enroll a total of four cohorts of two patients each. Initial 30-day safety and biomarker data is expected to be reported in mid-year 2021.The clearance of IND application from FDA follows receipt of Clinical Trial Authorization for PBGM01 from the U.K. in December 2020.

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Passage Bio on go with PBGM01 gene therapy trial in the U.S.