Let the iPSC experts generate high-quality, induced pluripotent stem cells (iPSC) and derived physiologically relevant cell line models from your healthy/disease human or non-human samples. If you do not have a starting sample, Applied StemCell (ASC) offers customizable sample collection services. With our optimized reprogramming protocols and comprehensive characterization services, we deliver iPSCs ideal for your basic research, drug discovery, drug screening, and preclinical cell regeneration projects:
- Highly optimized protocols with high reprogramming efficiency (>95% success rate)
- From healthy/diseased human or non-human samples
- iPSC generation from human PBMCs, fibroblast, HSC, MSCs, CD34+ cord blood, urine, and more; non-human PBMC and fibroblast
- Integration-free (episomal/mRNA/viral-based) or retroviral reprogramming
- Feeder-free protocols; optional feeder-dependent protocols available
- iPSCs characterized for morphology and pluripotency markers. Additional characterization such as G-banding, RT-PCR, STR profiling, directed differentiation is also available.
Not only can ASC further characterize your iPSCs, but our experts can also genetically engineer your iPSCs using CRISPR/Cas9 or TARGATT™ and differentiate the iPSCs to the cell type of your choice, including NK cells, T cells, astrocytes, cardiomyocytes, and more.iPSC Generation Service Categories
GMP Grade iPSC Services & Products
Allogenic & Immunocompatible iPSC Generation
iPSC Generation From Non-Human Species
Applied StemCell (ASC) has provided stem cell and genome editing services for over 12 years, and we have worked with researchers all across the globe to engineer over 500 unique cell line models. As one of the earliest providers of CRISPR/Cas9 genome editing services, ASC has the experience and optimized protocols for Rapid Automated Cell Line Editing (RACE™) in induced pluripotent stem cells (iPSCs)!
With our well-established high-throughput protocols, ASC's experts can produce any complex or mainstream genetic modification in your healthy or diseased iPSCs for your basic research, disease modeling, tissue engineering, regenerative medicine, or cell-based therapy research. Leverage our affordable, customizable iPSC genome editing service to obtain your genetically modified iPSCs in just a few weeks.
High success rate: >98% projects completed to customer’s specifications
ASC can genetically modify your healthy or diseased iPSCs; control lines are available
Single cell cloning (clonal isolation)
Homozygous or Heterozygous
Automated processes for consistency and high throughput scalability
Pluripotency maintained throughout genome editing process using high-end cell culture reagents and protocols
ASC is a one-stop-shop for all your iPSC service needs. We are one of the few providers of integrated upstream iPSC generation & downstream differentiation and assay development services. If you are looking to engineer iPSCs in a GMP setting, we invite you to read more about our new GMP-grade iPSC service offerings.
iPSC differentiated cell lines offer the convenience of cell line models with the biorelevance of primary cells but without the sourcing difficulties and lot-to-lot variability issues associated with primary human cells. Applied StemCell (ASC) offers comprehensive service for iPSC differentiation to lineage-committed cell types thereby expanding the scope of your research, drug discovery, or drug screening projects.
- Lineage-committed cell lines as determined by lineage-specific markers
- Differentiation from healthy/disease iPSC lines: customer cell lines, ASC’s control iPSC lines, or genetically engineered iPSCs
- High purity cell lines
- Non-integrating differentiation protocols
- Development of co-culture models with multiple lineages is available
- Fast turnaround time
Our experts can differentiate your iPSCs or you can select from our inventory of well-characterized iPSC control lines. ASC even offers upstream iPSC generation from human or non-human samples and high-throughput CRISPR genome editing services. To learn more about our affordable, custom iPSC services contact us today.
iPSC Differentiation Service Categories
Custom “Master” iPSC Cell Line Generation using TARGATT™ technology! The TARGATT™ Master Human iPSC Line contains an attP “docking site” at the hROSA26 safe harbor genomic locus. Any gene of interest can be stably inserted at the docking site using a corresponding “attB” containing TARGATT™ donor plasmid, with guaranteed gene expression. The efficiency of this phiC31 integrase-mediated recombination is up to 100% efficiency with drug selection and up to 30% without drug selection.
Our cell line model generation experts can also engineer your safe harbor locus knock-in iPSC lines using CRISPR/Cas9 for a multi-technology approach to generate a cell line model for your specific research needs.
The true potential of iPSCs and ESCs rest in its directed differentiation to different somatic lineages. As part of our comprehensive stem cell service platform, researchers can leverage our expertise in stem cell technologies and our cost-effective, reliable Stem Cell Differentiation Services for their developmental biology, disease mechanisms, and drug discovery research.
Neural Stem Cell Differentiation Categories
- Differentiate your patient-derived iPSCs into microglia and self-renewing, multipotent neural stem cells (NSCs) and further to neurons and glial cells.
- High differentiation efficiency and cell purity
- Cells fully characterized for microglia and NSC biomarkers
iPSC differentiation to Immune Cells, Blood Cells
iPSC-derived T cells, NK cells, HPCs CD34+, dendritic cells and others provide a next generation toolset for understanding cancer and disease pathology as well as developing successful immunotherapies (adoptive therapies like CAR-T, CAR-NK, TCR-T and more). Applied StemCell with its world-class comprehensive stem cell platform which includes iPSC generation, genome editing and differentiation, can differentiate your iPSCs into CD34+ hematopoietic lineage progenitor cells and further into high-quality lineage committed immune cells: CD8+, CD4+ T cells, CD34+ HPCs, NK cell, dendritic cells, monocytes, and more. These cells are ideal for immunotherapy research for cancer and immune disorders as well as to develop complex co-culture models to screen drugs and understand immune cell activation and function.
Applied StemCell provides top quality service for directed-differentiation of iPSCs into cardiomyocytes using our proprietary induction protocol and reagents. We deliver ready-to-use, functional cardiomyocytes that are desirable in vitro models for high content toxicity and drug screening, and cell regeneration research. They are a feasible and physiologically relevant alternative to embryonic stem cell, primary cells, and animal models.
We can generate differentiated cardiomyocytes from iPSC derived from healthy or disease patient samples.
Applied StemCell provides custom differentiation of your induced pluripotent stem cells (iPSCs) into high-quality hepatocytes for developing liver disease models, discovering therapeutic treatments, drug targets, and understanding drug induced hepatotoxicity.
Using our proprietary induction protocol and reagents, we can generate differentiated hepatocytes very efficiently and with high purity from your own healthy or disease iPSCs.
Our Hepatocytes Differentiation Service includes:
- Expansion of a host-derived iPSCs
- Progenitor cell differentiation
- Characterization of progenitor cells
- Terminal differentiation
- Characterization of Progenitor Cells/ Differentiated Cells by ICC (per marker)
Human iPSC-derived retinal pigment epithelium (RPE) provides a physiologically relevant cell line model to understand basic ocular biology and ocular diseases. ASC’s iPSC-derived RPE protocol provides high quality RPEs with expression of lineage-committed markers and functional phenotype such as phagocytosis, and by providing an unlimited source of these cells.
- RPE-like cells with typical cobblestone morphology and pigmentation
- High quality and purity cells (60-80% purity) expressing RPE-specific markers Bestrophin 1 (BEST1)/ RPE65
- iPSC reprogrammed from fibroblast/ PBMC/ CD34+ cord blood cells
- From healthy/ disease/ engineered iPSCs
- Optional! Control lines differentiated from “master” iPSC lines available
Applied StemCell offers iPSC differentiation to Myogenin+ and MHC+ skeletal muscle-like cells that form neuromuscular junctional models when co-cultured with motor neurons. We can also help you establish the co-culture model with your iPSC-derived cell lines using our high efficiency motor neuron differentiation protocol.
Monocytes, precursor cells of macrophages and dendritic cells, are immune cells that stem from bone marrow. These phagocytes use the bloodstream as a form of transportation and play an essential role in the innate immune system.
Macrophages are phagocytes that develop from monocytes. These immune cells engulf pathogens and contribute to the recruitment of other immune cells as part of the innate and adaptive immune systems.
Applied StemCell now offers comprehensive iPSC differentiation to lineage-committed cell types, including monocytes and macrophages. You provide ASC with your healthy, diseased, or edited iPSC line, and our scientist will follow our non-integration differentiation protocols to provide you with the iPSC-derived monocytes or macrophages that best fit your project needs in 2 months.
Photoreceptor cells, rods and cones, can be found in the retina and are sensitive to low and high levels of light, respectively. Rods and cones take light and generate signals that are sent to the brain where that information can be processed as part of the first steps in vision. Applied StemCell (ASC) uses non-integrating differentiation protocols to produces the photoreceptor cells that best fit your project needs. The lineage-committed photoreceptor cells can express photoreceptor-specific markers, including CRX, NR2, NR2E3, Tuhi1, Rhodopsin, and PPR6a.
To get you the iPSC-derived photoreceptor cells you need, you can send in your healthy or disease iPSC line, or you can request to use one of ASC’s control iPSC lines. If you would like to use a genetically engineered iPSC line for the differentiation of photoreceptor cells, ASC also offers high-throughput iPSC genome editing service.
With a fast turnaround time of 12-15 weeks, ASC is the one-stop shop you need to obtain high-purity photoreceptor cells. Applied StemCell now offers Adeno-Associated Virus (AAV) Infectivity assay service: serotype selection, promoter testing, and MOI optimization. Contact us today to learn more!
Photoreceptor Differentiation Service Can Include:
- iPSC Generation
- iPSC Characterization
- Photoreceptor Differentiation
- Photoreceptor Characterization
*Contact us today to speak with one of our experts who can help you design the project you need to drive your research forward.
Stringent molecular and functional assays are necessary to evaluate pluripotency and to rule-out genetic aberrations due to reprogramming and stress from in vitro culture in iPSC lines. Loss of genetic integrity can affect desired cellular phenotype and compromise interpretation and translation of results to a clinical setting.
ASC offers comprehensive services needed to completely characterize your human and mouse pluripotent stem cell lines (PSCs):
- Pluripotency and lineage-specific marker immunostaining
- Karyotyping (Chromosome counting, G-banding, array analysis)
- qPCR, RNA-seq
- Trilineage differentiation potential: embryoid body (EB) formation, qRT-PCR
- HLA typing, whole genome sequencing (WGS), single tandem repeat (STR) genotyping, copy number variations (CNV) and more
Our comprehensive start-to-finish ipsc and stem cell platform offers downstream services to advance your iPSC-based projects to the next stage. We offer preclinical CRO solutions for drug screening, protocol development for cell regeneration and adoptive transfer model, CAR-T research, universal and immunocompatible cell line, and more. We can customize every stage of your project and find the best fit to suit your research needs.
Applied StemCell has a staff of talented scientists that can help you with the more complex to the daily laboratory tasks required of iPSC cell culture. Give your staff the freedom to focus on other project, while Applied StemCell takes care of your cell culture needs.
We offer standard and customized cell culture services including mycoplasma testing.
- iPSC expansion services
- iPSC scale-up services
- Neural Stem Cells, Differentiated Cells scale-up services
- 3D-culture and morphology testing
Please contact us to discuss your needs with our technical service specialists.
3D culture systems for use with stem cells (iPSCs) are gaining popularity as matrix grown cells have longer pluripotency, better differentiation potential, proliferation and cellular function, and are more physiologically relevant in in vitro models.
Applied StemCell is a leader in stem cell technologies and provides custom service for 3D-iPSC culture of customer-provided iPSCs. We use our MyEZGel™ 3D-iPSC Matrix, a xeno-free nanopeptide hydrogel matrix that is specially formulated to provide an in vivo-like microenvironment to culture and generate spheroidal, physiologically relevant iPSCs.
- Cell-based assay development and validation
- Disease modeling
- Drug discovery and screening
- Tissue and organ engineering
- Cell replacement therapies
iPSC Derived NK, CAR-T/CAR-NK Cell Generation
As natural killer (NK) cell-based cancer immunotherapy research continues to grow, so has the number of potential chimeric antigen receptor (CAR)-NK therapies. Researchers continue to explore the cytotoxic capabilities of engineered NK cells, but several CAR-NK cell generation problems remain. For example, the CAR-NK cell development process takes several weeks, the patient’s NK cells may be weak and in a damaged state, or cancer cells may be created with random integration. Recent advancements in induced pluripotent stem cell (iPSC) technology have opened the door for the development of CAR-iPSCs that can be further differentiated into NK cells. These cells could potentially fix the current CAR-NK problems the research community is facing.
Applied StemCell (ASC) combined its optimized TARGATTTM gene editing and NK differentiation technologies to establish the TARGATTTM iPSC-iNK Platform. Our expert scientists can insert your specific CAR genetic material into our iPSCs at a safe harbor locus with an efficiency ~10x better than CRISPR and further differentiate your CAR-iPSCs to high-quality NK cells. Leverage our unlimited source of iPSCs and proprietary TARGATTTM technology to safely and efficiently produce the allogenic iNK cells you need to drive your research forward.
- >40% gene integration efficiency
- Site-specific knock-in
- NK cells derived from an unlimited source of iPSCs
- iPSC-iNK cell banks with a consistent manufacturing process
- Safe and efficient gene editing protocols
- Transfection by lipofectamine, eliminating viral manufacturing
- We generate truly off-the-shelf allogeneic therapeutic cells
As a CRO/CDMO service provider that continuously works to improve and expand its technology, services, and products, we hope to have our new cGMP TARGATT™ iPSCs available for purchase early next year. For now, we offer you fully characterized cGMP grade iPSCs from CD34+ cord blood. Contact us today to learn more.
We Understand Cell Lines! Leverage our expert, comprehensive CRISPR/Cas9 cell line modeling service for a stress-free research. With 1300+ unique cell line models engineered from >200 distinct mammalian cell lines, we can engineer perfectly suited cell line models for your research using highly optimized and efficient CRISPR/Cas9 genome editing strategies and protocols:
Cell Line Model Generation Categories
- Engineer a variety of mutations; or correct mutations, including fusion gene(s)
- Variety of cell lines: cancer, hard-to-transfect, blood lineage, stem cell lines and many more
- Start-to finish cell line generation workflow; optional downstream cell line validation
- Custom deliverables: homozygous/ heterozygous clones; point mutation with/without silent mutation
- >97% success rate; turnaround time as fast as 2 months
Cell Line Model
CRISPR Stem Cell
Knockout, SNV/Tag Knock-in & More
CRISPR/Cas9 Cell Line
Service - Hematopoietic Cells
(Jurkat and TF-1)
CRISPR Gene Fusion
Cell Line Generation
ASC is one of the earliest service providers of CRISPR/Cas9 technology, and has successfully delivered >500 CRISPR mouse models in as little as 3 months. Our animal model portfolio offers competitive pricing and turnaround times for generating CRISPR knockout, conditional knockout, locus-specific/ safe harbor knock-in, controlled gene expression and gene correction, and more.
Mouse Models Categories
- Most up-to-date CRISPR designing strategies and protocols
- 100% target-site cutting efficiency using optimized, proprietary gRNA validation methods
- Animal IP belongs to researchers
- Project management and scientific support to discuss your project needs and suitable strategic options to fit your budget
- NEW! Downstream electrophysiology and behavioral assessments for your mouse models
CRISPR Conditional Knockout
CRISPR Knockout, Knock-in,
Point Mutation Mouse Models
Homologous Recombination Conditional Knockout Mouse Model and Knock-in Mouse Models
Transgenic Mice Models
With the need to physiologically relevant animals models on the rise for more predictive research findings, genetically engineered rat models are becoming increasingly popular. Get ahead of your competition by leveraging Applied StemCell’s expertise in CRISPR/Cas9 and TARGATT™ genome editing technologies and animal models engineering to generate the ideal rat model to meet your specifications and budget.
Rat Models Categories
- Global leader in genetically engineered rat models (Nature article, “CRISPR mouse model boom, rat model renaissance”)
- Detailed discussion to understand project requirements, optimal project design for precise genetic modification and dedicated project management for timely updates
CRISPR Knock-In, Knock-Out,
Conditional Knock-Out Rat
Bacterial Artificial Chromosome (BAC) Knock-in and Conditional Knock-In Rat Models
Applied StemCell’s proprietary site-specific, integrase-based TARGATT™ technology can be used to generate stable, knock-in cells lines with large transgenes including stem cells, very efficiently and quickly. Knock-in is mediated by the integrase at a pre-engineered “docking site” in an intergenic, transcriptionally active genomic locus (safe harbor locus) for high level gene expression without disruption of internal genes. This technology allows only a single-copy integration with very high efficiency with or without clonal selection.
Use our TARGATT™ technology to generate your ”Master” cell lines, reporter/tag lines, for iPSC generation, conditional gene expression models and more.
Also, try our ready-to-use TARGATT™ Master Cell Lines to knock-in transgenes in your own lab.TARGATT™ Cell Line Categories
TARGATT™ High Resolution Protein Screening
ASC’s proprietary TARGATT™ knockin technology enables highly efficient and site-specific gene knockin mouse model generation. This technology uses serine integrase, PhiC31 (ΦC31) to insert any gene of interest into a preselected intergenic and transcriptionally active genomic locus that has been engineered with a docking site for stable, site-specific transgene integration.
- High-efficiency insertion (up to 65%)
- Site-specific into a genetically active safe harbor locus
- Large fragment knock-in (up to 22 kb)
- Single copy in an active locus: avoid gene silencing and genomic instability.
- High level, stable gene expression
- Germline transmitted F1 mice in 5-8 months
Mouse models: Gene overexpression, conditional expression, humanized gene knockin, gene knockdown, reporter genes
TARGATT™ Fast & Site-Specific Knock-in Mouse Service Workflow
Applied StemCell’s proprietary TARGATT™ technology enables generation of physiologically relevant transgenic rat models suitable for a variety of applications including reporter gene expression, gene knock-down, conditional gene expression and disease models. This technology uses the Phic31 integrase to mediate an irreversible integration of large transgene(s) into a preselected, safe harbor locus with very high efficiency.
- Site-specific, single copy transgene integration overcomes challenges associated with random integration
- TARGATT™ knock-in rats in 6-9 months
- Direct microinjection of the TARGATT™ reagents into rat zygotes
Using the TARGATT™ technology, ASC has also developed Neural Specific Cre-Rat Lines in a Sprague Dawley rat background.
TARGATT™ Fast & Site-Specific Knock-in Rat Service Workflow
TARGATT™ CHO Cells for Recombinant Protein and Antibody Bioproduction! Applied StemCell’s TARGATT™ Master CHO cell lines leverage the site-specific gene integration capacity of serine integrase, phiC31 to knockin transgene(s) into transcriptionally active genomic hotspots, such as the H11 or the proprietary ASC2 (A2) safe harbor locus. These master cell lines enable highly efficient and rapid gene, and high levels recombinant protein/ antibody expression.
Our Master TARGATT™ CHO cell lines can exceed the capabilities of the traditionally made CHO antibody production cells and offers a platform for affordable and feasible bioproduction for antibodies and other recombinant proteins for companies and projects of all sizes.
TARGATT™ Rabbit for Biopharming and High-Yield Recombinant Protein and Antibody Production
Animal bioproduction uses transgenic animal mammary gland as a bioreactor for the production of recombinant proteins. Animal bioproduction, compared to CHO bioproduction, has the advantages of lower upfront and maintenance costs; is easy to contain, control and transport; involves faster development processes and no scale-up issues; and has a unique low-cost bulk holding stage (frozen milk). However existing technological methods such as random integration to create transgenic animals have many limitations. Applied StemCell has overcome these limitations with our proprietary TARGATT™ technology to reproducibly, and consistently, control the knock-in locus, copy number and expression of the target protein in transgenic animals.
Applied StemCell (ASC) is a fast-growing gene and cell therapy CRO that continuously works to expand its technologies, services, and products to address the current and future needs of the industry and research community. ASC understands that monoclonal antibodies (mAbs) are essential for research and development, so we established and optimized a single B cell antibody discovery platform that can be used to generate high-quality rabbit or alpaca antibodies. Choose from our selection of unique rabbit strains/lines, or ask us about our customized gene knockout rabbit. You can select our classical or accelerated immunization protocol based on your timeline constraints. Once the immunization process is complete, our team conducts single B cell cloning and delivers 40 ELISA positive recombinant mAbs and a data report. You can screen and select your clones of interest, or ASC can complete the screening process for you (*additional fees apply). After antibody selection, our scientists sequence and produce the elected antibodies.
During your free consultation, our experts can help you design a flexible project outline that captures all of your requirements. Once the project design is finalized, you send us your antigen and we take care of the rest. ASC will have your rabbit monoclonal antibodies in just a few weeks.
Advantages of ASC's Single B Cell Antibody Discovery Platform
- Rabbit Monoclonal Antibodies with Subnanomolar Affinity
- HyImmune Rabbits typically produce 3× antigen specific B cells, to increase the antibody development rate
- Unlimited Supply: Customer owns IP
- More Antibodies: >0.5mg for each antibody selected
- Convenient: Antibodies can be delivered with or without luciferase
- Alpaca Antibody Service Available - Inquire
- Affordable Pricing
- Fast turnaround time
Unique Animal Strains & Lines
Optimized Immunization Protocol
Rapid Amplification of Single B Cell mRNA
Efficient Mammalian Cell Antibody Expression
Applied StemCell provides lentiviral and retroviral custom virus packaging services for your CRISPR/Cas9 components, CAR-T expression vectors and other transgenes, for efficient delivery into cell lines (such as hard-to-transfect cell lines, primary cell lines) as well as direct injection into animal models.
- Rapid, custom lentivirus or retrovirus virus packaging
- Ready-to-transduce viral particles provided to you
- High quality, high titer viral particles in 10 days
- VSV-G pseudotyped viruses that exhibit broad tropism across a range of cell types
- High titer amplification of viruses, up to 10^9 infectious units per ml
- Accurate viral titers quantified by qRT-PCR
- Confirmation by DNA sequencing
- Large scale production, clonal expansion and cryopreservation
Applied StemCell is one of the earliest licensees and provider of CRISPR/Cas9 genome editing services. We are experts in designing and cloning components for CRISPR-based gene editing in cell lines and animal models using benchmark strategies and optimized protocols. Our scientists have designed > 1000 CRISPR projects for researchers worldwide, and we use the latest sequencing and next generation sequencing (NGS) protocols to validate your gRNAs and to ensure high efficiency gene modification in your gene of interest.
We offer CRISPR/Cas9 vector design, construction and gRNA validation for:
- Cell line gene modification
- Microinjection into embryos for animal model generation
- Virus packaging
We also offer comprehensive custom CRISPR cell line and mouse/ rat model generation services.
Applied StemCell has extensive expertise in anything DNA-related. We can develop the best strategy to tackle your cloning projects, and find solutions to all your technical problems.
- Comprehensive services for genome engineering for in vivo and in vitro applications
- Restriction fragment cloning
- Design and validation of CRISPR/Cas9 components: gRNA design and in vitro validation by next generation sequencing (NGS)
- Gene targeting vectors for homologous recombination
- Design and construction of vectors for transient mammalian transgene expression
- Bacterial artificial chromosome recombineering for large fragment insertion
- Design of RNAi and inducible vectors, gene tagging, and site-directed mutagenesis
- Vector design for generation of random transgenic cell line and animal models
Bacterial Artificial Chromosomes (BACs) are ultra-low copy vectors that can hold up to 300 kb of genomic fragments, making them ideal vectors for introduction of entire genes including the regulatory regions for disease modeling with transgenic animals. BACs are traditionally difficult to modify with restriction enzymes and ligases because of their large size. Applied StemCell uses homologous recombination to introduce precise changes into BACs for your specific experimental needs. Virtually any desired modification can be introduced into a BAC, including insertion of reporter genes, point mutations, Lox-STOP-Lox conditional modifications, and more.
Our custom BAC services include:
Applied StemCell can also help you create transgenic rats using BACs.
As a long-standing leader in genetically engineered animal models, Applied StemCell also offers a comprehensive custom research solutions platform for downstream assays in mouse and rat models. Our state-of-the-art vivarium is equipped with automated cages & devices for behavioral assessments, sample collection and in vivo measurements (ECG, EEG) of the animals. We have a multidisciplinary team of expert scientists who can design a comprehensive project plan to fit any requirement/stage of your research pipeline and for drug discovery and screening.
- Designing and engineering research animal disease models
- Adoptive transfers/transplantation
- In vivo functional screening assays
- End-of-study in vitro assays
ASC offers a full suite of comprehensive services tailored towards your preclinical-stage cell and gene therapy (CGT) projects. Leverage our fully integrated service platform starting with in vitro model generation and cell-based assays, followed by custom animal models and in vivo assays for target validation for early go-no-go decisions of your biotherapeutics. We have a unique team of scientists with multidisciplinary expertise in CRISPR/ genome engineering technologies, stem cell biology, immuno-oncology, gene therapy models, and viral vectors to plan and execute every aspect of your CGT projects. We can generate in vitro and in vivo models, design viral vectors (AAV, lentivirus) and develop proof-of-concept and target validation assays specific to your therapeutic pipeline needs.Discovery: Preclinical Cell & Gene Therapy Services Categories
Cell Line Model Validation
Animal Model Validation
Cell and Gene Therapy (CGT) drugs have their unique mechanism of action and require a more tailor-made approach for drug development and regulatory approval. Starting from custom cell line and animal model generation for the targeted disease, ASC’s multidisciplinary team of experts can develop preclinical assays to determine efficacy, pharmacodynamic, dose-ranging studies, biodistribution, and develop other IND-enabling assays to measure potency (AAV potency, DNA/ RNA/ protein levels) and toxicity of the drug in multiple species samples including NHPs, immunogenicity of the drug, and more. We will work with you every step of the way including pre-IND meetings with the FDA/ respective regulatory bodies to develop the right assay matrix for your CGT candidates.
Detailed Chemistry, Manufacturing & Control (CMC) information submission is a critical part of your IND-submission irrespective of the class of therapeutic under investigation in order to validate the identity, purity, potency, viability, titers, potency, cell dose of your drug candidate. ASC offers expert CMC consultation and assay support for your investigational biotherapeutics: Lot release specifications, stability analysis, potency analysis at DNA/ RNA/ protein levels, titration, cGMP cell line characterization for cell therapies, we offer you a support and resources to navigate the guidelines provided by the FDA or other regulatory authorities to enable regulatory compliance.
Make Informed Go-No-Go Decisions Early in Your Drug Development Process!
Leverage Applied StemCell’s unique expertise in CRISPR/Cas9 and induced pluripotent stem cell (iPSC) technologies to engineer predictive and reliable in vitro disease models as an alternative to animal models for preliminary drug screening.
Our ISO:9001 certified service platform offers full flexibility in choosing assay modules with a wide range of functional endpoints for early-stage in vitro screening of preclinical drug candidates. We have a comprehensive cell-based test battery from which you can choose assays for efficacy, safety or target discovery that suit your therapeutic pipeline. We provide the scientific expertise, accurate and efficient screening to help you make informed decisions about your small/ large molecules early in your drug development process.