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One patient died in a Phase II/III study of gene therapy type IIIA adhesive polysaccharose storing disorder.

 Last Update: 2020-10-17
 Source: Internet
 Author: User

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Lysogene disclosed Thursday that one patient in the Phase II/III AAVance study died and is undergoing gene therapy LYS-SAF302 to treat type IIIA adhesive polysaccharose storage disorder (MPS IIIA).
Lysogene's share price fell as much as 19 percent on the news, and the cause of death is not yet known, but there is no evidence linking the death to LYS-SAF302.
LYS-SAF302, a gene therapy for MPS IIIA developed by Lysogene, is a gene therapy mediated by adenopathic viruses (AAVs) that aims to replace defective SGSH genes by delivering functional SGSH genes through AAV.
three elements of research gene therapy (LYS-SAF302) for MPS IIIA are: vector: AAVrh10 initiater: CAG delivery gene: complete SGSH gene Lysogene said it would study the remaining 18 patients "strictly in accordance with the research program."
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