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Axovant Sciences Licenses Two Gene Therapy Programs from University of Massachusetts for Rare and Fatal Neurodegenerative Genetic Disorders

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Axovant Sciences Licenses Two Gene Therapy Programs from University of Massachusetts for Rare and Fatal Neurodegenerative Genetic Disorders

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  • Axovant acquires WW development and commercialization rights for AXO-AAV-GM1 and AXO-AAV-GM2 Program and will make payments to University of Massachusetts
  • The focus of the agreement is to develop functional copies of HEXA- HEXB & GLB1 genes encoding the certain enzymes to treat Gm1 Gangliosidosis- Tay-Sachs and Sandhoff Diseases
  • AXO-AAV-GM1 program is initiated for GLB1 gene using AAV vector for activating β-gal enzyme activity to treat GM1 gangliosidosis- with its expected results in H2’19
  • AXO-AAV-GM2 is programmed for HEXA and HEXB genes developed via AAVrh8 vectors to activate Hex A enzyme activity for both Tay-Sachs and Sandhoff diseases- with its expected results in H1’19
Ref: Axovant Sciences | Image: Twitter

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