Fresh from the spectacular failure of its dementia drug, nelotanserin, which has been pushed to the scrap heap along with its much-hyped Alzheimer’s asset, Axovant $AXON is moving on fast at the behest of its deal hungry patron Vivek Ramaswamy.
On Thursday, the company said it had struck a deal to license the global rights to develop and market two gene therapy programs designed to address two progressive and fatal pediatric lysosomal storage disorders: GM1 gangliosidosis and GM2 gangliosidosis – also known as Tay-Sachs and Sandhoff diseases – from the University of Massachusetts Medical School.
The two assets add heft to the London-based company’s existing pipeline of gene therapies. A mid-stage trial testing its existing gene therapy for Parkinson’s is set to read out data in March.
Meanwhile, a program evaluating the newly licensed AXO-AAV-GM2 in GM2 gangliosidosis is already underway, with initial data expected in the first quarter of 2019. The first GM1 gangliosidosis patient to be dosed with AXO-AAV-GM1, the other asset, will likely occur next year in an investigator-initiated clinical trial conducted at the NIH.
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