The financing will be used to further develop its pipeline of 17 adeno-associated virus (AAV)-based gene therapy product candidates in partnership with the University of Texas Southwestern Gene Therapy Program. The company also has options to acquire four additional programs across three distinct franchises, including neurodegenerative diseases, neurodevelopmental disorders, and genetic forms of epilepsy.

Clinical studies for the treatment of GM2 Gangliosidosis are slated for later this year, followed by treatments for Rett syndrome, SLC6A1 haploinsufficiency disorder, and SURF1 deficiency. Investigational new drug applications are expected to be filed for these product candidates by the end of 2021.

Additionally, financing, which is led by Fidelity Management and Research Company, will be used to build a commercially scalable good manufacturing practices manufacturing facility.

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