GMP & RUO Grade iPSC Gene Editing & Differentiation Services

Name: GMP & RUO Grade iPSC Gene Editing & Differentiation Services
Brand: Applied Stem Cell

Use Applied StemCell's (ASC) new GMP facility, comprised of five cleanrooms equipped with cutting-edge technology, to develop the next generation of iPSC-derived therapeutics. We have dedicated cleanrooms for induced pluripotent stem cells (iPSC), mesenchymal stromal cells (MSC), chimeric antigen receptor T-cells (CAR-T), T-cell receptor-engineered T-cells (TCR-T), natural killer (NK) cells, CAR-NK cells, and viral vectors. Explore our end-to-end manufacturing service options or optimize your GMP SOP with our GMP master cell bank or high-quality starter options.

GMP & ROU iPSC Gene Editing

Engineer mainstream point mutation and knockout cell lines or safely and efficiently generate large complex knock-in cells for further differentiation and assay development.

Advantages of ASC’s Gene Editing Technology

• TARGATT site-specific knock-in of any gene of interest up to 20kb (iPSC-GMP-TARGATT)

• SSelect for large cargo insertion with double-digit efficiency

• ceBE-X for base editing for knockout and point mutation with zero off-target events

• Optimized CRISPR/Cas9 protocols for high-throughput editing

• GMP and Research Use Only services

Pluripotency is maintained throughout the gene editing process, and medium to high expression levels have been observed.

GMP & ROU iPSC Differentiation

Differentiate normal or genetically modified iPSCs into natural killer (NK) cells, cardiomyocytes, photoreceptor cells, neural progenitor cells (NPCs), astrocytes, and many more cell types. Receive

robust, lineage-committed cells identified by lineage-specific markers that have undergone standard characterization.

Comprehensive Differentiation Service

• Differentiation from healthy or disease iPSC lines: customer cell lines, ASC’s control iPSC lines, or genetically engineered iPSCs

o Off-the-shelf iPSCs: (1) GMP iPSCs (2) GMP Matching iPSCs (For Research Use Only) (3) Knock-in ready TARGATT-iPSCs (Also available for CIRM awardees)

• High-purity cell lines

• Non-integration differentiation protocols

• Co-culture model development

• Cell expansion and banking (24/7 monitoring)

• GMP and Research Use Only Services

The differentiated high-purity cells express high levels of their specific cell markers: (1) NK cells (≥ 80% purity) express CD56 and CD45, (2) cardiomyocytes (≥80% purity) express cTnT, (3) photoreceptor cells (≥90% purity) cells express CRX and NR2E3, (4) NPCs (≥95% purity) express Nestin, and (5) astrocytes (≥95% purity) express GFAP and s100beta.

Product Specifications

iPSC Generation Service Timeline 2-3 months

CRISPR Gene Editing Service Timeline (1) Knockout, 6-8 weeks (2) Point mutation, 6-8 weeks (3) Knockin, 10-15 weeks

TARGATT Gene Editing Service Timeline 3-5 months

NK Cell Differentiation 2 months

Cardiomyocyte Differentiation 2 weeks

Photoreceptor Differentiation 3 months

NPC Differentiation 3 weeks

Astrocyte Differentiation 2 months

MSC Banking ~ 2 .5 weeks

MSC Expansion ~2 weeks

About Applied Stem Cell

Applied Stem Cell (ASC) is a leading cell and gene therapy CRO/CDMO in California, USA. ASC offers comprehensive and customized cell and gene solutions. Its core iPSC and genome editing (CRISPR and TARGATT™) technologies facilitate site-specific, large cargo (up to 20kb) transgene integration and the development of allogenic cell products. It has a Drug Manufacturing License from the California Department of Public Health, Food and Drug Branch (FDB) and Quality Management System (ISO 13485 certified). With a new state-of-the-art facility and cGMP-compliant protocols, ASC has entered the manufacturing space, now offering cell banking, iPSC generation, genome editing, iPSC differentiation, and cell product manufacturing.

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