Gene Therapy Development

Classified as an advanced therapy medicinal product (ATMP), gene therapy is subject to specific regulations and requires sophisticated testing technologies to bring to market. From regulatory expertise to a comprehensive portfolio of discovery through manufacturing services, you’ll find everything you need to make your gene therapy a reality at Charles River.

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Significant advances in technology like CRISPR/Cas 9 have paved the way for gene therapy as a viable approach to treating disease. As a unique biologic product, however, gene therapies demand equally custom solutions to ensure their safety, efficacy and stability. With an industry-leading portfolio of services and regulatory expertise, Charles River can help you create and execute a robust program that carries you from early research to the clinic and beyond.

Our global network of facilities offers gene therapy testing services where you are, with sites in the US, Canada, France, Ireland, and Germany; and, with 70 years of success in bringing medicines to market, you can trust our team to help you reach your goals. Whether you’re looking for insight on approvals, stand-alone testing services, or full program support, fuel your gene therapy development with Charles River.

Webinar Series: Platforms for Gene Therapy Characterization


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Enhance your existing analytical methods and explore innovative, novel tools to support safe and effective gene therapy development.

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Browse our Gene Therapy Testing Services and Resources

Genetic model creation services. Engineering human targets? Ease the integration of genetically created models with your downstream discovery using our genetic model creation and contract breeding services. If a genetic model exists, our decades of breeding experience can get you the cohorts needed.

Donated human cells. Validate your target with optimal starting materials. Our HemaCare and Cellero line of products ensures a consistent, reliable and quality-controlled human donor cells.

Immunodeficient mouse models. Test your therapy in models that effectively host human tissue.

CRISPR/Cas 9 gene editing. Use CRISPR technology as a cost-effective method for cellular assay development in a variety of early discovery applications.

Antisense oligonucleotide screening. Accelerate your hit ID and optimization of the optimal ASO with our in vitro screening platform.

Bioinformatics Services. Learn how to get the most out of your existing data from next generation sequencing to mining big data to discover precision therapies.

Successful development of an ATMP, such as a gene therapy, involves a program that is exclusive to a drug candidate and patient population, while encompassing broad regulatory expectations. Meet the unique and diverse challenges of gene therapy with a team approach that includes experts in pharmacology, toxicology, immunology, bioanalysis, molecular biology, guided by extensive regulatory insight.

We can help you to identify key questions to establish proof of concept (e.g., efficacy or gene modulation), evaluate biodistribution, and outline goals for pilot and definitive safety assessment. However, study design, and subsequent conduct, is only a first step. In a true collaborative effort, we’ll work closely with you and your team to establish a comprehensive and sound nonclinical data package and providing scientific and regulatory support throughout the development process.

Case Study: Evaluating Multiple Vectors in a Single Eye

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A custom surgical technique to create four independent subretinal injection sites within a single eye for a gene vector.

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Trust our team’s robust, reliable methods for clinical sample analysis. From the start of your nonclinical program through subsequent testing, our scientists collaborate as part of your clinical team. This approach results in robust and reliable methods for clinical sample analysis, which, when combined with data management, enables us to deliver high-quality on-time clinical data to support your drug approval.

Our well-established experience in sample management and kit assembly for Phase I through III clinical trials worldwide is reflected in the 1,300+ sample kits we produce per month. We also offer extensive biomarker testing services, including clinical efficacy/PD biomarkers that allow you to confirm target engagement, and improve your chance of reaching Phase III clinical trials. Our CLIA certification enables us to develop novel biomarkers for use in diagnostic testing, post-market patient safety monitoring, and companion diagnostic testing.

Pharmacodynamic (PD) Biomarkers Video:

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Discover the advantages of using clinical/efficacy PD biomarker in this brief interview with Russell Garland, our Group Leader of Analytical Services.

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Our comprehensive package of GMP CMC manufacturing and testing services supports your gene therapy development with more than 20 years of experience of cell bank manufacturing and associated biosafety tests and characterization studies. We’ll help you mitigate risks and overcome challenges of development while complying with the required regulatory standards.



Inaccurate identification of microorganisms, within your facility, can halt product production during parametric release. AccuGENX-ID® utilizes Sanger sequencing technology, our BacSeq bacterial identification of the 16S rRNA gene and FunITS fungal identification of the ITS2 region. These services offer reliable and accurate results in as little as six hours.

Similar to our AccuGENX-ID methods for bacterial and fungal identification, our AccuGENX-ST sequence typing uses standard molecular biological methods to characterize your isolates to the strain level. The resulting DNA sequences, or sequence types, are compared to others in a database and the relationships, similarities and/or differences, are displayed in a phylogenetic tree. Providing accurate strain level characteristics allows you to definitively track the source of contamination and/or verify and track known production strains within your facility.

Accugenix Resources:

Endosafe® cartridge technology offers features that will particularly benefit Cell and Gene Therapy labs due to the nature and volume of samples produced by cell and gene therapies. Endosafe cartridge technology can be used with our handheld, portable rapid endotoxin testing system, the Endosafe® nexgen-PTS™ or our multi-cartridge system, Endosafe® nexgen-MCS, for results in 15 minutes. Because the archived standard curve is included in the LAL endotoxin testing cartridge, the nexgen-PTS or nexgen-MCS is easy to use and technician variability is reduced meaning lower retest rates saving you time and resources. Endosafe cartridge technology offers swift results, which allows cell and gene therapy firms to release life-saving product quickly.

Endosafe Resources:

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Frequently Asked Questions (FAQs) about Gene Therapy

  • What is a gene therapy?

    Genes are responsible for creating the proteins the control cellular processes. In many diseases, genetic instructions are missing or defective. Scientists first identify the implicated gene in patient cells then create and modify a copy so that it functions properly. The gene “therapy” is then transmitted back to the patient via vector. Once delivered to patient cells, the gene therapy provides instructions for creating the intended protein, correcting the defect.

  • What is an ATMP (advanced therapeutic medicinal product)?

    An advanced therapeutic medicinal product, or ATMP, categorizes a class of therapies derived from genes, somatic cells (cells that do not produce eggs or sperm), or tissue products. The development of ATMPs is governed by a specific set of regulations that differ from other kinds of therapies.

  • What is the difference between a gene and cell therapy?

    Both considered an advanced therapeutic medicinal product, or ATMP, a gene therapy uses genetic material to modify the function of patient cells in the treatment of inherited or acquired disease, while a cell therapy involves the infusion or transplantation of whole cells (e.g., bone marrow transplant or CAR-T).

  • What is a viral vector?

    A viral vector is a gene therapy delivery system. Typically created from a virus called an adeno-associated virus (AAV), naturally occurring entity known not to cause illness in people, the AAV vector is an ideal vehicle to transmit gene therapies to the body.

  • What are the most common viral vectors for gene therapies?

    Typically, gene therapy development uses adeno-associated viruses (AAV), but the following viral vectors can be used (amongst others not listed here which are less common):

    • Retrovirus
    • Lentivirus
    • Adenovirus