• CRO Services

CRO Services

With > 12 years experience in genome editing and stem cell technologies, ASC provides ISO:9001 quality, customizable solution-oriented services for advancing preclinical assay development and drug screening for cell and gene therapy pipelines and bioprocessing/ bioproduction:

  • Preclinical proof-of-concept cell line and animal models
  • IND-enabling preclinical assay development, validation, and testing: potency, safety/toxicity, efficacy
  • Assays include dose ranging, biodistribution and efficacy studies in cell line and animal models for your drug discovery and screening applications
  • Consultation for cell and viral vectors manufacturing, analytical methods for drug release (assay development, validation, testing).

Customize each part of your project to fit the stage of your research.

CRO Services Categories

Discovery: Model
Generation & Validation

Reliable, custom cell line and animal models, AAV/ lentiviral vector designing, and validation assays for your early-stage cell/ gene therapy projects.

Discovery: Model
Generation & Validation

IND-enabling: Pharmacodynamics,
Efficacy Assay Development

Custom assay development for determining proof-of-concept for your CGT, efficacy, pharmacodynamics, biodistribution, and toxicity.

IND-enabling: Pharmacodynamics,
Efficacy Assay Development

CMC Support: Cell and Gene Therapy Development

Preclinical and non-clinical CMC support for investigational cell and gene therapy products.

CMC Support: Cell and Gene Therapy Development

 

FAQ

 

Support Materials

 

Application Notes

 

Technical Details

ASC’s 12+ years experience in animal and cell line model genetic engineering and stem cell technology is a powerful resource you can leverage to advance your gene and cell therapy pipeline. Our mission is to equip biotechnology companies with a series of optimized tools for supporting therapeutic target discovery, drug screening, preclinical assay development, preclinical therapeutic development, and bioprocessing/ bioproduction.

Preclinical & Biologics Analysis: From Cell and Animal Modeling to Assay Development

Cell line models

Mouse models

Rat models

Vector building

 

IND-Enabling

Proof-of-concept

Dose ranging 

Biodistribution

In vivo assay development

Efficacy

Toxicity

Biologic analysis

Potency assay

Reference standards

DNA/RNA/Protein Analysis

Titration

CMC consultation

Cell line characterization

IND-Enabling Studies: We can help you navigate FDA’s increasingly regulated gene and cell therapy requirements from pre-IND, preclinical safety, and efficacy requirements all the way to your IND filing. Our multidisciplinary think-tank will work you every step of the way to provide you with scientific consultation for study design development, regulatory compliance, safety and efficacy endpoint determination as well as preclinical custom services to engineer, characterize, validate and test suitable in vitro and in vivo models for target engagement and efficacy assays for your therapeutic candidates.

We can customize every part of your project to fit the stage of your research. Be it cell replacement therapy or gene therapy (AAV-based), we can help. Below is a selected list of assays/services that we can help you with:

In vitro and in vivo model generation and evaluation

Product/vector/construct optimization

Proof-of-concept in vivo functional efficacy studies

Cell viability & cell-based assays

AAV serotype infectivity in cells and animals

AAV in vivo biodistribution and shedding

Potency assay development

(DNA/ RNA/ Protein)

Pilot toxicity studies

Bioprocessing/ bioproduction

We operate under strict ISO guidelines and document control process. All data is secured with a multi-layer approach to enforce cyber security.

Contact us for more details into the assays or for a free & confidential consultation to discuss your requirements.


Project Workflow: Initiation through Completion 

CRO Project Workflow

Committed to Excellence:

MEET INDUSTRY REGULATORY COMPLIANCE REQUIREMENTS WITH HIGH QUALITY PRODUCT AND SERVICES

ISO 9001:2015      Cert # 1100091
ISO 13485:2016     Cert # 1100090

Biosafety Level 2 Laboratory

AAALAC-accredited facilities

Studies Performed in a Manner Consistent with Principles of GLP

FDA 21 CFR Part 58

QA Review of Protocols

Case Studies

 

Publications

Cell and Gene Therapy Applications:

  • Chen, H., Shi, M., Gilam, A., Zheng, Q., Zhang, Y., Afrikanova, I., ... & Chen-Tsai, R. Y. (2019). Hemophilia A ameliorated in mice by CRISPR-based in vivo genome editing of human Factor VIII. Scientific reports9(1), 1-15.

Other CRO Services:

  • Mace, E. M., Paust, S., Conte, M. I., Baxley, R. M., Schmit, M., Mukherjee, M., ... & Akdemir, Z. C. (2019). Human NK cell deficiency as a result of biallelic mutations in MCM10. bioRxiv, 825554.
  • Baskfield, A., Li, R., Beers, J., Zou, J., Liu, C., & Zheng, W. (2019). Generation of an induced pluripotent stem cell line (TRNDi004-I) from a Niemann-Pick disease type B patient carrying a heterozygous mutation of p. L43_A44delLA in the SMPD1 gene. Stem cell research37, 101436.
  • Hong, J., Xu, M., Li, R., Cheng, Y. S., Kouznetsova, J., Beers, J., ... & Zheng, W. (2019). Generation of an induced pluripotent stem cell line (TRNDi008-A) from a Hunter syndrome patient carrying a hemizygous 208insC mutation in the IDS gene. Stem cell research37, 101451.
  • Cheng, Y. S., Li, R., Baskfield, A., Beers, J., Zou, J., Liu, C., & Zheng, W. (2019). A human induced pluripotent stem cell line (TRNDi007-B) from an infantile onset Pompe patient carrying p. R854X mutation in the GAA gene. Stem cell research37, 101435.
  • Yang, S., Cheng, Y. S., Li, R., Pradhan, M., Hong, J., Beers, J., ... & Zheng, W. (2019). An induced pluripotent stem cell line (TRNDi010-C) from a patient carrying a homozygous p. R401X mutation in the NGLY1 gene. Stem cell research39, 101496.
  • Baskfield, A., Li, R., Beers, J., Zou, J., Liu, C., & Zheng, W. (2019). An induced pluripotent stem cell line (TRNDi009-C) from a Niemann-Pick disease type A patient carrying a heterozygous p. L302P (c. 905 T> C) mutation in the SMPD1 gene. Stem cell research38, 101461.
  • Huang, W., Xu, M., Li, R., Baskfield, A., Kouznetsova, J., Beers, J., ... & Zheng, W. (2019). An induced pluripotent stem cell line (TRNDi006-A) from a MPS IIIB patient carrying homozygous mutation of p. Glu153Lys in the NAGLU gene. Stem Cell Research, 101427.
  • Sundararaj, K. P., Rodgers, J., Angel, P., Wolf, B., & Nowling, T. K. (2020). Neuraminidase activity mediates IL-6 production through TLR4 and p38/ERK MAPK signaling in MRL/lpr mesangial cells. bioRxiv.
  • Li, R., Baskfield, A., Lin, Y., Beers, J., Zou, J., Liu, C., ... & Zheng, W. (2019). Generation of an induced pluripotent stem cell line (TRNDi003-A) from a Noonan syndrome with multiple lentigines (NSML) patient carrying a p. Q510P mutation in the PTPN11 gene. Stem cell research34, 101374.
  • Li, R., Pradhan, M., Xu, M., Baskfield, A., Farkhondeh, A., Cheng, Y. S., ... & Rodems, S. (2018). Generation of an induced pluripotent stem cell line (TRNDi002-B) from a patient carrying compound heterozygous p. Q208X and p. G310G mutations in the NGLY1 gene. Stem Cell Research, 101362.
  • Poli, M. C., Ebstein, F., Nicholas, S. K., de Guzman, M. M., Forbes, L. R., Chinn, I. K., ... & Coban-Akdemir, Z. H. (2018). Heterozygous Truncating Variants in POMP Escape Nonsense-Mediated Decay and Cause a Unique Immune Dysregulatory Syndrome. The American Journal of Human Genetics, 102, 1-17. https://doi.org/10.1016/j.ajhg.2018.04.010
  • Vozdek, R., Long, Y., & Ma, D. K. (2018). The receptor tyrosine kinase HIR-1 coordinates HIF-independent responses to hypoxia and extracellular matrix injury. Sci. Signal.11(550), eaat0138.
Have Questions?

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