Understanding of Disease Mechanism:
New insights into a disease process by bioinformatics approaches utilizing genomic information associated with the disease. The target genes involved in specific conditions can be identified by sequencing, qPCR, and Whole Genome Screening using CRISPR gRNA.
Target Gene Validation:To validate targets, researchers use techniques such as cell-based models, phenotypic models, IHC, FACS , and WB for target engagement to demonstrate that the target is involved in the progression of a given disease.
Lead Identification & Optimization
Candidate Identification, Screening and Optimization:
Lead identification is to design and create a vector that can modify, replace, or Inactivate the selected target. Researchers can conduct screening experiments to identify possible vectors for drugs. Once vectors have been identified, they need to be optimized to maximize the GOI expression and delivery efficacy and minimize immune reaction for safety. The vectors optimization can prevent off-target binding, making them less prone to interact with molecules other than the target.
Preclinical Development and IND Application:
Once a lead vector is identified and optimized, the preclinical phase of drug development begins with in
vivo research on non-human primates for efficacy, toxicity, and pharmacokinetic (PK) information. These
studies are conducted by scientists in vitro and in vivo with a wide range of dosages. After preclinical
testing, results are reviewed and the decision is made on whether the drug should proceed to human trials.
To progress to human clinical trials, the FDA requires extensive testing and data, including Animal study data and toxicity, Manufacturing information(CMC), Clinical protocols, and Data from any prior human research to be submitted in the IND application for approval.
Monitoring the Therapy Safety and Effect in patients :
This phase assesses drug safety and efficacy on healthy volunteers and patients. It helps researchers evaluate the safety and pharmacokinetics, absorption, metabolic, and elimination effects on the body and any side effects for safe dosage ranges. The complexity of clinical trial design, associated costs, and implementation issues may affect trials carried out during this phase.
Explore our technologies and learn more about Gene Therapy Solution. Find the information and resources you need by browsing through our educational material.
Josh Wang, Ph.D.
R&D Sr. Scientist Genscript USA Inc.
Senior scientist at GenScript USA Inc.
Shondra M. Pruett-Miller
PhD Director, St. Jude Children’s Research Hospital Comprehensive Cancer Center
James Chon Ph.D
Field Application Scientist Genscript USA Inc.