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Rare Disease

Neurogene launches to tackle rare neurological disorders

by Lisa Jarvis
February 15, 2019 | A version of this story appeared in Volume 97, Issue 7

 

Rare-disease-focused Neurogene has debuted with $68.5 million in its first formal round of financing from investors that include Samsara BioCapital, EcoR1 Capital, and Redmile Group. The biotech firm is developing gene therapies for rare neurological diseases and plans to use the proceeds to fund clinical studies, invest in technology, and build a viral vector production site. Neurogene, which partners with academic scientists and patient advocacy groups, is initially pursuing gene therapies for two lysosomal storage disorders and a rare form of the sensory disorder Charcot-Marie-Tooth.

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