Gene Therapy Analysis Services
Gene Therapy Analysis Services
Gene therapy is a complex class of advanced therapeutics that requires rigorous analytical characterization, process control, and regulatory-compliant testing to ensure product identity, purity, safety, and potency throughout development and manufacturing. With over two decades of experience supporting gene therapy development, we offer comprehensive analytical services tailored to facilitate the development and regulatory approval of various gene therapy modalities. Below, you’ll find an overview of each type of gene therapy we support, along with the analytical and bioanalytical services we offer to support preclinical and clinical studies, as well as CMC.
Our Expertise
We specialize in quality control, product release, and bioanalytical testing, to support all phases of gene therapy products from development through manufacture. Our contributions to preclinical and clinical studies have been instrumental in securing numerous successful gene therapy approvals.
Preclinical/Clinical Testing (GLP or non-GLP)
Vectors
Experience
Viral Vector
Since the early 1990s, we have been actively supporting gene therapy utilizing viral vectors such as AAV, adenovirus, lentivirus, and vaccinia virus.
Plasmid
Our team excels in providing comprehensive analytical support of plasmid gene therapy, from vector design and construction through preclinical testing and clinical study support.
Microbial Vector
We have experience supporting clients utilizing microbial vectors, such as Salmonella, to deliver therapeutic genes to target cells or tissues.
In Situ sgRNA/mRNA in LNPs
We leverage advanced qPCR, ddPCR, and NGS tools to deliver innovative analytical and bioanalytical methods supporting in situ sgRNA and mRNA therapies.
mRNA in LNPs
Since 2015, we have been a pivotal partner to one of the leading mRNA vaccine developers, offering extensive expertise in supporting mRNA vaccine and therapeutics.
Analytical Support Through All Stages of Product Lifecycle
Phase
Technologies
Applications
Development
qPCR, ddPCR, PCR, Sanger Sequencing, NGS, HPLC, Flow Cytometry, cloning, CRISPR/cas9
Preclinical
CMC Support
qPCR, ddPCR, PCR, Sanger Sequencing, NGS, Flow Cytometry, Compendial Assays
- Identity assays
- Potency assays
- Vector genome integrity testing
- Stability testing
- Replication competent virus testing
- Copy number variation (CNV) testing
- Adventitious agents testing
- Sterility testing
- Mycoplasma testing
- Endotoxin testing
- Bioburden testing
- Residual DNA assays
- Residual host cell protein assays
- Other release assays
Regulatory Guidance
This final guidance provides industry with answers to frequently asked questions (FAQs) and commonly faced issues that arise during the development of cellular and gene therapy (CGT) products, spanning regulatory review, chemistry, manufacturing, and controls (CMC), pharmacology/toxicology, clinical, and clinical pharmacology topics. This guidance finalizes the draft version issued in...
We, the FDA, are issuing this draft guidance to assist you (manufacturers, applicants, and other stakeholders) in developing human gene therapy (GT) products incorporating ex-vivo and in vivo genome editing (GE) of human somatic cells (GE products). Specifically, this guidance reflects FDA’s current thinking on the type of prior knowledge...
For a high-level overview of this guidance document, please watch this recorded webinar featuring Kimberly Schultz, PhD (Office of Therapeutic Products, FDA) and LCDR Jessica Dunn, PhD (Office of Compliance and Biologics Quality, FDA). This guidance describes how FDA applies flexibility to the chemistry, manufacturing, and controls (CMC) requirements for...
