Gene therapy drugs represent a molecular form of precision treatment achieved through the integration of functional components such as nucleic acid vectors and targeting elements. Drawing upon extensive expertise in gene therapy characteristics, InnoStar has systematically addressed research challenges and established a comprehensive non-clinical evaluation system that meets global regulatory standards. With full capability to support multi-center international submissions, InnoStar is dedicated to delivering efficient and reliable R&D services that accelerate innovative drug development. we has successfully supported over 100 gene therapy development projects, including multiple "first-in-class" initiatives with global and domestic innovation significance.

InnoStar Gene Therapy Solutions

Early Screening Strategies for Gene Therapy Products

- Vector construction and optimization: Screening high-efficiency viral (e.g., AAV serotypes) and non-viral (e.g., LNP) delivery systems for target genes
- Transfection efficiency screening: Evaluating gene delivery and expression efficiency in target cells
- Species specificity analysis: Assessing vector compatibility with animal model (e.g., mice, cynomolgus monkeys) cellular receptors
- In vitro functional validation: Testing gene editing/expression effects on target cell function (e.g., knockout, protein overexpression)
- Preliminary safety assessment: Analyzing potential cytotoxicity or immune activation responses in vitro

Non-Clinical Pharmacokinetics

- Vector biodistribution and clearance: Investigating tissue-specific accumulation and elimination kinetics of viral/non-viral vectors to evaluate targeting efficiency
- Serum stability testing: Examining structural integrity and degradation rates in animal/human serum to optimize delivery systems
- Gene expression kinetics: Monitoring spatiotemporal expression patterns of target mRNA/protein to determine duration of effect
- Vector residue analysis: Quantifying residual vector DNA/RNA or viral particles in biological matrices to assess long-term exposure risks

Non-Clinical Safety Evaluation

- Safety pharmacology: Evaluating potential effects on critical systems (cardiovascular, respiratory, CNS)
- General toxicology: Investigating short-/long-term toxicity of vectors and transgenes with histopathological correlation
- Immunotoxicity studies: Detecting anti-vector antibodies (e.g., AAV capsid) and cytokine storm risks while assessing immunosuppression potential
- Reproductive toxicity testing: Monitoring placental transfer and germline effects to evaluate embryonic gene transfer risks
- Carcinogenicity assessment: For integrating vectors, examining potential oncogene activation risks from exogenous DNA insertion
- Off-target editing screening: Employing whole-genome sequencing to evaluate CRISPR-related off-target risks
- Genetic toxicity testing: Assessing genotoxic potential of gene therapy products
- Local tolerance testing: Evaluating injection/infusion site (e.g., brain, liver) inflammatory responses and tissue damage

Clinical Bioanalysis

- Biomarker analysis: Correlating gene expression levels with efficacy/toxicity to optimize dosing and vector selection
- Vector quantification: Utilizing qPCR/digital PCR platforms for nucleic acid quantification in biological matrices
- Transgene expression analysis: Detecting target protein concentration and functionality via ELISA/Western blot
- Immunogenicity assessment: Employing ELISA/neutralization assays to evaluate anti-vector and anti-transgene antibodies
- Off-target monitoring: Tracking potential off-target sites and mutation frequencies using NGS technologies

Project Experience

- Cumulative completions: 100+ new drug R&D projects
- Regulatory milestones: 10+ NMPA IND approvals, nearly 10 FDA IND approvals, with successful China-U.S.-Europe multi-submission cases

Partner with InnoStar for your gene therapy program to navigate the path from discovery to regulatory approval with scientific excellence and operational confidence.