AXGT - Axovant Gene Therapies candidate nabs Rare Pediatric Disease tag for nerve cell disorder

The FDA grants Rare Pediatric Disease designation to Axovant Gene Therapies' (AXGT) AXO-AAV-GM1, an AAV0-based gene therapy for the GM1 gangliosidosis, a rare inherited disorder characterized by the progressive destruction of nerve cells (neurons) in the brain and spinal cord.Rare Pediatric Disease designation provides for the issuance of a priority review voucher following FDA approval. The voucher can be used for accelerated approval of a future application or it can be sold to a third party.A Phase 1/2 clinical trial is in process.Quant rating is Neutral.

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Axovant Gene Therapies candidate nabs Rare Pediatric Disease tag for nerve cell disorder