Solutions Introduction

GenScript offers a full range of services and products for Gene Therapy. From Target Discovery to Lead Identification and Optimization to Pre-clinical and Clinical. We can support Gene Therapy companies throughout the development and manufacturing process with our RUO, pre-clinical, and clinical-grade services and products.

Gene Therapy Solution Workflow

Lead Identification & Optimization 2

Target Discovery

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.

Target Discovery

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.

Lead Identification & Optimization

Pre-clinical Studies

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.

Pre Clinical Study

Clinical Studies

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.

Gene Therapy Resources

Explore our technologies and learn more about Gene Therapy Solution. Find the information and resources you need by browsing through our educational material.

  • Target Discovery

  • Lead Optimization

  • Pre-Clinical Study

  • Clinical Development

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