Gene Therapy for Cardiovascular Diseases

Cardiovascular disease (CVD) is the number one cause of death worldwide, and traditional treatments can relieve symptoms but not reverse the disease process. Research has identified important roles in the pathogenesis of CVD, including both monogenic and polygenic mechanisms and innovative gene therapies are being developed to correct the pathogenic variants (PVs) that lead to genetic diseases. CD Formulation is a biotechnology company specializing in the development of gene therapy formulations and is committed to helping researchers accelerate the development of gene therapy formulations through innovative technologies and providing more reliable research protocols.

Advantages of Gene Therapy for Cardiovascular Diseases

• Precise targeting, long-lasting cure. A single treatment can last for years or even a lifetime, avoiding long-term drug dependence. For example, AAV vector-delivered gene drugs can be stably integrated into cardiomyocytes, releasing therapeutic proteins continuously.
• Breaking through the bottleneck of difficult-to-treat diseases. For end-stage heart failure and intractable angina, which are ineffective with drugs, gene therapy offers new hope by promoting collateral circulation or enhancing myocardial function.
• Controllable side effects. The risk of off-targeting is dramatically reduced through tissue-specific promoters and novel vector design (e.g., engineered AAV variants).

Main Applications of Gene Therapy for Cardiovascular Diseases

Heart failure

Advances in gene therapy research for heart failure include gene therapy targeting myocardial repair, i.e., delivery of the cBIN1 gene to cardiac cells via adeno-associated virus (AAV), which achieved a 30% improvement in cardiac pumping function and reversed myocardial remodeling in a porcine model, with a significant increase in survival. It also enhances myocardial contractility by the delivery of protein phosphatase 1 inhibitor (I-1c).

We provide the following services to support heart failure gene therapy research.

• Customized vector development. Provide AAV serotype screening and delivery system optimization for targeting myocardium.
• Gene editing technology platform. Develop gene therapy programs targeting myocardial repair based on CRISPR-Cas9 or base editing technology.
• Preclinical validation. Evaluate efficacy and safety through large animal models (e.g., pigs).

Coronary artery disease

Gene therapy research in CAD includes regulation of lipid metabolism genes, such as inactivation of the hepatic PCSK9 gene by base editing and editing of the ANGPTL3 gene for lipid lowering. There is also delivery of the vascular endothelial growth factor (VEGF) gene via AAV to promote neovascularization of ischemic myocardium and improve perfusion.

We offer the following services to support gene therapy research in coronary artery disease.

• Liver-targeted delivery system. Liver-specific gene editing using LNP or AAV vectors for precise regulation of lipid metabolism.
• Multi-target combination therapy development. Design synergistic therapeutic programs by combining PCSK9, ANGPTL3, and other targets.
• Biomarker validation. Provide marker testing for lipoprotein(a), inflammatory factors, and other markers.

Cardiovascular genetic diseases

Cardiovascular genetic diseases are caused by single gene mutations, such as hypertrophic cardiomyopathy (MYH7 mutation). Hypertrophic cardiomyopathy caused by MYH7 or TNNT2 mutations has been studied by correcting the causative gene through in vivo editing, and animal models have shown reversal of cardiac hypertrophy. Functional recovery was achieved in a genetic cardiomyopathy model using single-base editing to turn off mutant gene expression.

We offer the following services to support gene therapy research for cardiovascular genetic diseases.

• Mutation-specific vector design. Customized CRISPR or base editing tools based on genetic test results.
• Fast Track Support. Utilization of biomarkers to shorten the clinical trial cycle.
• Long-term safety assessment. Provide multi-year follow-up data management to fulfill regulatory requirements for gene therapy by regulatory agencies.

Our Capabilities of Gene Therapy Formulation Development in Cardiovascular Disease

• Innovative technologies. We focus on developing optimized gene delivery vectors to improve the precision and efficacy of gene delivery.
• Rigorous quality control. We conduct strict quality control and thorough analysis of the gene therapy agents we develop to ensure product safety and efficacy.
• Comprehensive collaboration. To date, we have made significant contributions and achievements in translating cutting-edge research into practical applications through close collaboration with industry experts.

Explore Our Gene Therapy Formulation Development for Cardiovascular Diseases

CD Formulation specializes in the development of gene therapy formulations. Relying on our advanced technology platform and years of service experience, we provide reliable solutions for the development of cardiovascular gene therapy formulations for our clients. If you are interested in us, please feel free to contact us.