Preclinical Research On Gene Therapy

We provide compliant preclinical research services for gene therapy to support candidate drugs in meeting regulatory requirements.

The regulatory guidelines for the toxicology and safety pharmacology studies necessary for advancing gene therapy into human clinical trials are continuously evolving. Unlike other therapeutic categories, such as small molecules or biologics, the preclinical research requirements for gene therapy are tailored to the specific characteristics of the therapy. Furthermore, gene therapy encompasses a variety of approaches, including in vitro gene editing and in vivo viral and non-viral vector therapies.

Types of gene therapy vectors include:

Viral (lentivirus, adenovirus, AAV)
Naked DNA, nanoparticles, RNA

Biological BSL-3 and Gene Therapy Preclinical ResearchUnder Biosafety Level 3 (BSL-3) conditions
Preclinical Research On Gene Therapy

Provide emergency priority access to candidate drug services in a biosafety level laboratory.

Plasmid DNA:Circular DNA molecules can be engineered to carry therapeutic genes into human cells.

Viral Vectors:Viruses have a natural ability to deliver genetic material into cells, making them useful for some gene therapy products. Once viruses are modified to remove their infectious capabilities, they can serve as vectors to carry therapeutic genes into human cells.

Bacterial Vectors:Bacteria can be modified to prevent them from causing infectious diseases and then used as vectors to deliver therapeutic genes into human tissues.

Human Gene Editing Technologies:Gene editing aims to disrupt harmful genes or repair mutated genes.

Patient-Derived Cell Gene Therapy Products:Cells are extracted from the patient, genetically modified (often using viral vectors), and then returned to the patient.