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Axovant licenses Tay-Sachs, Sandhoff disease therapy programs from university

Axovant Sciences Ltd. said it acquired a license to two gene therapy programs from the University of Massachusetts Medical School, or UMMS.

The exclusive worldwide license covers AXO-AAV-GM1 and AXO-AAV-GM2 — potential treatments for nerve diseases classified under GM1 and GM2 gangliosidoses, respectively. GM2 gangliosidosis is also known as Tay-Sachs and Sandhoff diseases.

Axovant Sciences, through its unit Axovant Sciences GmbH, will be responsible for the development and commercialization of AXO-AAV-GM1 and AXO-AAV-GM2. The London-based developer of neurological and psychiatric treatments paid $10 million up front for the license, which it can sub-license.

UMMS will also receive up to an additional $24.5 million based on the achievement of certain development and regulatory milestones, as well as up to an additional $39.8 million based on the achievement of certain commercial milestones. UMMS will receive tiered mid-single digit annual sales royalties for the licensed products, as well as a mid-single digit to mid-single teen percentage of revenues from Axovant Sciences' sub-licensees of AXO-AAV-GM1 and AXO-AAV-GM2.

GM1 and GM2 gangliosidoses, which are due to genetic defects, progressively destroy nerve cells in the brain and spinal cord. Children with these diseases experience cognitive impairment and paralysis, with a life expectancy of two to four years. There is currently no cure for GM1 and GM2 gangliosidoses, which occur about once in every 100,000 and 180,000 live births worldwide, respectively.

The licensed therapies work by delivering functional copies of defective genes to restore the function of otherwise impaired enzymes.

AXO-AAV-GM1's first patient is expected to be dosed in the first half of 2019, with initial data from its clinical program expected in the second half. Continued enrollment for the program is expected throughout 2019. AXO-AAV-GM2's first patient has been dosed, with initial data from its clinical program expected in the first quarter of 2019. Enrollment for its multi-subject clinical trial is also expected to start in 2019.