Combination gene therapy

Pre-existing and treatment-induced antibodies can block AAV-based gene therapy and prevent repeat dosing. Our approach is being developed to address these barriers through targeted antibody removal, aiming to make gene therapy safer and more accessible.

On this page:

Understanding gene therapy and its challenges

Gene therapy is an innovative approach that aims to treat genetic disorders by introducing healthy copies of a gene into a patient’s cells. To deliver these genes, scientists often use harmless carriers called vectors, which are often based on adeno-associated viruses (AAV). These vectors act like delivery vehicles, carrying the therapeutic gene to the right cells. However, many people naturally have antibodies against them, which can block treatment from working. After the first dose, the body often produces even more antibodies, making repeat treatments extremely difficult. Overcoming this immune barrier is essential to unlock the full potential of gene therapy.

The antibody barrier to gene therapy

Antibodies against AAV vectors are a major limitation for gene therapy programs. They can exclude patients from treatment and prevent redosing after the first infusion. Addressing this challenge is critical for expanding patient access and enabling long-term treatment strategies.

Targeted antibody depletion for gene therapy

Unlike conventional blood purification or systemic immunosuppression approaches, our technology is being developed to selectively deplete antibodies that block gene therapy while preserving the broader immune system. The goal is to provide a controlled, application-specific solution that supports first-dose eligibility and future redosing strategies in patients requiring gene therapy.

Partnering across the development journey

Selective antibody depletion has the potential to add value at every stage of gene therapy development. hemotune works with partners from early discovery through clinical development and post-market strategies, aiming to address antibody-related challenges and support innovative treatment approaches.

Explore feasibility and accelerate animal studies by enabling dosing in NHPs with pre-existing anti-AAV antibodies.

Develop strategies intended to expand patient eligibility, enable redosing, and support approaches that may reduce vector dose requirement

Plan for scalable solutions to integrate antibody depletion into long-term treatment strategies.