ASC Awarded $1.3 mil USD from NIH for TARGATT™ Gene Editing | ASC

Applied StemCell Awarded $1.3 mil USD from NIH for TARGATT™ Gene Editing

8/16/17 MILPITAS, Calif.--Applied StemCell, Inc. (ASC), a leading gene-editing company focused on the development of enabling tools and curative medicines using TARGATT™ and stem cell technologies, has just been awarded close to $1.3 million US dollars in a small Business Innovation Research (SBIR) grant from the NIH. The grant award will support the establishment of a Cre/LoxP rat repository for enabling better rat models of human disease. Using the company’s proprietary TARGATT™, site-specific gene insertion technology, ASC plans to generate twenty (20) novel Cre-driver lines, of which seventeen (17) lines will be specific for neural lineage expression, two (2) models will be specific for cardiovascular expression, and one will allow for ubiquitous Cre expression. Using its in-licensed CRISPR technology, ASC will also generate conditional LoxP-flanked (floxed) alleles to pair with these Cre-driver rats to make inducible and tissue-specific models. To our knowledge, this represents a first effort in establishing such a Cre resource for rat model generation. The lack of such an effort in the past has largely been due to technical difficulties and high costs associated with traditional methods of rat line engineering. In contrast, tremendous effort and resources have been put into establishing Cre mouse lines, which has enabled diverse and important scientific discoveries. We anticipate that this new rat model resource will have an even larger impact on scientific and biomedical advances, particularly in the areas of neurophysiological, behavioral, and cardiovascular studies.

ASC’s Strategy and Pipeline

ASC’s strategy is to utilize its core strengths to expand its IP portfolio and develop a strong therapeutic pipeline based on its patented gene editing technologies. The company’s expertise in utilizing genome editing for both animal and cell model generation, as well as its portfolio of improved genome editing technologies, make it uniquely poised to develop transformative solutions to pressing medical challenges.

About Applied StemCell

Applied StemCell, Inc. is a leading stem cell and gene-editing company focused on the development of products and therapeutics that are enabled by its proprietary gene editing platform technologies TARGATT™ and CRISPR/Cas9. For more information, please visit www.appliedstemcell.com.

Contact:

Maki Ogawa

Marketing Director , Applied StemCell, Inc.

408-773-8007

maki.ogawa@appliedstemcell.com