RTTNews | Oct 9, 2020 07:20AM EDT

07:20 Friday, October 9, 2020 (RTTNews.com) - Axovant Gene Therapies Ltd. (AXGT) said Friday that it has received Rare Pediatric Disease Designation from the U.S. Food and Drug Administration for AXO-AAV-GM1. It is an AAV9-based gene therapy delivered through a single intravenous administration that is in Phase 1/2 development for GM1 gangliosidosis.

In addition, AXO-AAV-GM1 has Orphan Drug designation and is the only gene therapy in clinical development for both infantile (Type I) and juvenile (Type II) GM1 gangliosidosis, the company said in a statement.

The company said it is on-track to report 6-month Stage 1 data from the low dose juvenile cohort (Type II) by the fourth quarter of 2020, and expects to initiate the high dose cohort which includes infantile (Type I) and juvenile (Type II) patients in the second half of 2020.

GM1 Gangliosidosis is a progressive and fatal pediatric lysosomal storage disorder caused by mutations in the GLB1 gene leading to impaired production of the -galactosidase enzyme.

Read the original article on RTTNews ( https://www.rttnews.com/3135133/axovant-gene-gets-rare-pediatric-disease-designation-for-axo-aav-gm1-for-gm1-gangliosidosis.aspx)

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