Applied StemCell

Applied StemCell, Inc. (ASC) is a genome engineering and cell therapy services company headquartered in Milpitas, California. It positions itself as a CRO/CDMO for the research community and biotechnology industry, supporting the discovery, development and manufacturing of cell and gene products. Its service lines cover genome engineering (using CRISPR, Mad7 and the proprietary TARGATT large-cargo knock-in platform), induced pluripotent stem cell (iPSC) services spanning iPSC generation, gene editing and differentiation into target cell types, and cGMP cell banking and expansion performed in its California facility with customization for size, concentration and in-process/release testing.

Alongside services, ASC sells catalog products including TARGATT knock-in kits, cGMP-grade iPSCs, iPSC lines and iPSC-differentiated cells. Featured offerings include ActiCells GMP hiPSCs, described as covered by a US FDA Drug Master File with karyotype-normal genomic stability and stable transgene expression after editing and differentiation, and a TARGATT Hypo hiPSC Knock-in Kit that is hypoimmunogenic via a B2M/CIITA double knock-out, allows further knock-ins from the H11 safe harbor locus, and is offered license-ready. Earlier company descriptions also list custom gene editing in mice and rats, patient-relevant cell models, iPSC-based preclinical neurotoxicity and efficacy screening, Cas9-expressing cells, iPSC-derived neuronal lineage cells and cardiomyocytes, bioproduction in rabbits and cell lines, and stem cell-qualified MEF cells and FBS. A diagnostics division, AccuRef Diagnostics, offers engineered reference standards for cancer diagnostics, including 270 isogenic cell lines and more than 2,600 singleplex and multiplex dPCR reference standard products in formats such as genomic DNA, FFPE cell blocks, slides and scrolls.

Founding story

Applied StemCell was founded in 2008 by Dr. Ruhong Jiang (Founder, CEO and President) and Dr. Ruby Yanru Chen-Tsai, who were motivated by Shinya Yamanaka's iPSC reprogramming research to build a company providing genome modification and stem cell toolkits for animal and cell line modeling. Chen-Tsai had established the Stanford University Transgenic Research Center in 1996 after postdoctoral training at Stanford School of Medicine and directed it for 16 years; she served as a founder-consultant in ASC's early years before joining full time as CSO and Senior VP in 2012.

Business model

ASC operates a services-plus-products model: fee-for-service and CRO-based custom projects in genome engineering, iPSC work and animal/cell model generation; cGMP contract development and manufacturing (CDMO) including GMP cell banking and expansion; and a catalog of research and GMP-grade reagents, kits, cell lines and diagnostic reference standards. Its TARGATT platform is also offered on a licensing path, with products marketed as "license-ready with a clear IP path to commercialization."

Revenue derives from custom CRO/CDMO project fees (gene editing, iPSC generation and differentiation, GMP cell banking and manufacturing) and sales of catalog products such as knock-in kits, iPSC lines, differentiated cells, MEF cells/FBS and diagnostic reference standards, plus technology licensing of TARGATT.

Traction

The Milpitas site reports a track record of over 15 years and thousands of completed projects for companies, academic institutions and standard-setting bodies, roughly 10,000 sq ft of CRO/research laboratory space, five qualified ISO 7 cGMP cleanrooms with space planned for five more EMA Annex 1-compliant rooms, ISO 13485 certification since 2017 and a California Department of Public Health license. Catalog traction cited includes stem cell-qualified MEF cells and FBS referenced in more than 60 peer-reviewed publications and an AccuRef Diagnostics catalog of over 2,600 products. Research programs have been supported by NIH SBIR awards (including grant 1R43GM108071-01A1 for TARGATT rats and a further SBIR for TARGATT rabbits) and CIRM funding for iPSC work.

Latest developments

In October 2023 QHP Capital acquired Applied StemCell for undisclosed terms. The company's current commercial focus includes ActiCells GMP hiPSCs and the TARGATT Hypo hiPSC Knock-in Kit for allogeneic cell therapy development, and it reports plans and available space to expand cGMP operations with five additional EMA Annex 1-compliant cleanrooms plus further QC and materials management space.