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.
Browse our Gene Therapy Testing Services and Resources
- Research Models and Services
- Safety Assessment
- Manufacturing Services and Products
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.
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.
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.
- Cell therapy CDMO services
- Plasmid DNA CDMO services
- Viral vector CDMO services
- Molecular cloning services
- Viral vector packaging services for research discovery
- Plasmid and viral vector products
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):