ASC Awarded ~USD 2 mil NIH grant for Advancing its TARGATT Technology for Therapeutic Gene Editing | ASC

August 13, 2020 06:00 AM Eastern Daylight Time MILPITAS, Calif.--(BUSINESS WIRE)--Applied StemCell, Inc. (ASC), a leading gene editing company focused on the development of enabling tools for cell and gene therapy has been awarded close to $2.0 million US dollars in a Small Business Innovation Research (SBIR) grant from the NIH.

This fast track Phase I/II grant award will support Applied StemCell (ASC) to advance and improve the efficiency of its proprietary TARGATT™ integrase technology for human gene therapies (referred to as the hTARGATT™) in a three part objective: (1) Develop and improve the efficiency of the TARGATT™ technology in human cell lines including primary cells and stem cells; (2) Develop therapeutic transgenes and demonstrate optimal integration efficiencies as a functional demonstration of the platform’s utility for ex vivo therapeutic applications; (3) Differentiate the above mentioned stem cells to desired somatic lineages and demonstrate regulatory induction of the transgenes. This study will provide useful insight into the applicability of the hTARGATT™ Gene Therapy platform and build the foundation for its use in gene therapy for potentially ten thousand monogenic human diseases.

www.appliedstemcell.com

Contacts:

Maki Ogawa

Marketing Director, Applied StemCell, Inc.

408-773-8007

maki.ogawa@appliedstemcell.com