Gene Therapy Development Services

Gene therapy targets and regulates brain tumor-associated genes using viral vectors or non-viral delivery systems. Alfa Cytology provides development services for brain tumor gene therapies based on AAV/lentiviral vectors and CRISPR technology.

Introduction to Gene Therapy for Brain Tumors

Gene therapy employs targeted delivery systems-such as adeno-associated viruses (AAVs), lentiviruses, or lipid nanoparticles-to precisely introduce therapeutic genes (e.g., tumor suppressor genes, immune regulators, or oncolytic virus genes) into brain tumor cells. By integrating blood-brain barrier penetration techniques like focused ultrasound or receptor-mediated transport can modulate tumor proliferation, apoptosis, and immune evasion pathways. This field encompasses strategies such as CRISPR gene editing,and RNA interference to silence oncogenic targets that selectively kill tumor cells.

Fig 1. Graphic of viral vector gene therapy agents. (SHAH S, et al., 2025)

Gene Therapy Development for Brain Tumors

Disclaimer: Alfa Cytology focuses on providing preclinical research service. This table is for information exchange purposes only. This table is not a treatment plan recommendation. For guidance on treatment options, please visit a regular hospital.

Our Services

Alfa Cytology assists clients in developing gene therapies by leveraging an AAV/CRISPR gene-editing platform combined with multimodal delivery technologies to efficiently overcome the challenges of the blood-brain barrier and tumor heterogeneity. We offer a comprehensive, one-stop solution-including vector design, in vitro and in vivo model validation-to help overcome bottlenecks in the research and development of brain tumor gene therapies.

AAV Gene Therapy Development Services

Alfa Cytology provides end-to-end AAV gene therapy development services, including vector design and optimization, scalable production, and comprehensive preclinical and clinical support to accelerate your therapy from concept to market.

DNA-Based Gene Therapy Development Services

By introducing normal DNA fragments to replace or repair defective or mutated genes, DNA-based gene therapy aims to fundamentally correct genetic diseases. Functional genes are delivered into patient cells via viral vectors to restore normal function.

RNA-Based Gene Therapy Development Services

RNA-based gene therapy uses RNA molecules to regulate gene expression. By interfering with messenger RNA (mRNA) processes-such as using small interfering RNA (siRNA) or mRNA vaccines-it suppresses the production of harmful proteins or promotes the expression of functional ones.

Workflow of Gene Therapy Development

Target Screening and Mechanism Research

Based on multi-omics data-such as single-cell sequencing and epigenetic analysis-we identify brain tumor-specific targets (e.g., oncogenic gene mutations, immunosuppressive factors).

Vector System Design and Optimization

We select viral vectors (AAV, lentivirus) or non-viral vectors (lipid nanoparticles) and enhance their targeting specificity through engineering modifications (e.g., directing them to tumor cell surface receptors). We also optimize their payload capacity to deliver therapeutic agents like CRISPR/Cas9, shRNA, or oncolytic genes.

In Vitro Evaluation

Utilizing patient-derived brain tumor organoids to simulate the tumor microenvironment, we assess vector delivery efficiency, gene editing specificity, and anti-tumor activity. This enables us to optimize the precision of therapeutic strategies.

In Vivo Evaluation

In immune-humanized mouse models or orthotopic glioma models, we validate the in vivo efficacy and durability of gene therapy. This is achieved through live imaging, tumor volume monitoring, and survival analysis, providing comprehensive insights into therapeutic outcomes.

Alfa Cytology possesses extensive expertise and capabilities in the research and development of gene therapies for brain tumors, providing comprehensive, one-stop development services that cover every stage from initial discovery to clinical application. If you encounter any challenges or difficulties during your project, please do not hesitate to contact us-we are more than happy and fully prepared to assist you in overcoming any obstacles to ensure the success of your endeavors.

Reference

1. SHAH S, GREEN J, GRAFF S A, et al. Gene Therapy for Glioblastoma Multiforme [J]. Viruses, 2025, 17(1).