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