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.
Gene Sequencing and Analysis
Target engagement and Biomarkers Analysis
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.
Rational Design of AAV Capsid
GOI Expression Optimization
sgRNA/HDR payload Design and Screening
mRNA Design and Screening
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
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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.
IND
Phase I
Phase II
Phase III
BLA
Commercial
Most Popular Products
CRISPR Gene Editing
Antibodies
Protein Electrophoresis and Blotting
Molecular Biology
Stable Cell Lines
Cell Therapy
Diagnostics
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