Gene Therapy Development

We offer a complete scientific pathway for the development of your gene therapy. This covers activities across the development cycle ranging from plasmid and vector production, through generation of custom genetic models, and surgical expertise to clinically appropriate administration. Beyond this, our scientific experience and collaborative structure allow us to ensure appropriate program design at all points during progression to first in human trials. We can work with your internal teams to define critical development milestones and support you in making important decisions along the way.

Explore how we can tailor our gene therapy services to meet your needs and help you achieve your program goals.

How can we help

DNA sequence

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, gene therapies demand custom solutions to ensure safety, efficacy and stability. With an unsurpassed portfolio and regulatory track-record, we can help create and execute a robust program that carries you from early research to market.

Our extensive network of facilities offers global and local solutions, with sites in the United States, Canada, United Kingdom, France, Ireland, and Germany. Given our track record of 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 products or testing services, or full program support, you can count on us to fuel your gene therapy development.

DNA SciFi Helix

Webinar: High Quality Plasmid DNA: A Case for Phase Dependent Approaches to Manufacturing

Determine the optimal phase dependent approach to plasmid supply for product developers, such as HQ (High Quality) plasmid DNA.

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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.

Photo of an eye.

Case Study: Evaluating Multiple Vectors in a Single Eye

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.

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Pharmacodynamic (PD) Biomarkers Video:

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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The Disruptors: Conversations in Science

Sanath was determined to track down the resources needed to repurpose a drug for his son Raghav's ultra-rare disease. Find out how his repurposed drug platform is now giving hope to other rare disease patients.
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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