[{"orgOrder":0,"company":"Oryzon Genomics","sponsor":"Kabuki Syndrome Foundation","pharmaFlowCategory":"D","amount":"$1.0 million","upfrontCash":"Undisclosed","newsHeadline":"Oryzon Announces $1 Million Grant From Kabuki Syndrome Philanthropists to Support a Precision Medicine Phase I\/II Trial With Vafidemstat","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"SPAIN","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Oryzon Genomics"},{"orgOrder":0,"company":"Viralgen","sponsor":"Sio Gene Therapies","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Axovant and Viralgen Sign Strategic Gene Therapy Development and Manufacturing Partnership","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"SPAIN","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Viralgen"}]
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The company will use the grant to start Phase I/II trial, named HOPE, which will be a multicenter, double-blind and placebo-controlled trial to explore the safety and efficacy of vafidemstat in improving several impairments described in KS patients.
Axovant will have access to manufacturing resources for Axovant’s AAV-based gene therapy programs, AXO-AAV-GM1 for GM1 gangliosidosis and AXO-AAV-GM2 for GM2 gangliosidosis with sufficient capacity to support ongoing development and eventual commercialization.