DiNAQOR Unveils Breakthrough Catheter-Based Platform for Precision Gene Therapy Delivery
核心洞察
DiNAQOR (搜索) and academic partners published a landmark study in JACC: Basic to Translational Science demonstrating a first-in-class percutaneous catheter-based closed-loop perfusion platform for organ-isolated delivery of advanced therapies.
The platform achieved up to 69,000-fold higher local payload concentration and 75-fold increase in target-organ transduction efficiency compared to conventional intravenous administration in large-animal models.
Mayo Clinic experts endorsed the technology as a "game-changer" that closes the gap between promise and precision in gene delivery by enabling scalable, outpatient-compatible precision delivery across multiple organs.
DiNAQOR (搜索), a Swiss health tech company, has announced the publication of a groundbreaking study in JACC: Basic to Translational Science that describes a revolutionary catheter-based platform for precise organ-targeted delivery of advanced therapies. The technology addresses one of the most critical challenges in gene and cell therapy (搜索) development: safe and efficient in vivo delivery while minimizing systemic exposure.
Revolutionary Closed-Loop Perfusion Technology
The newly developed closed-loop loco-regional perfusion (LRP) platform represents a paradigm shift in therapeutic delivery. Unlike traditional systemic administration approaches, the fully percutaneous, catheter-based system enables complete isolation of an organ's circulation, allowing therapeutic payloads to be delivered locally under tightly controlled physiologic conditions.
In large-animal translational models, the platform demonstrated remarkable performance metrics. The system achieved up to a 69,000-fold higher local payload concentration and up to a 75-fold increase in target-organ transduction efficiency compared with conventional intravenous administration. Simultaneously, the technology dramatically reduced off-target exposure while preserving organ function and procedural safety.
Professor Maximilian Y. Emmert, M.D., Ph.D., Professor of Cardiothoracic and Vascular Surgery at Charité – Universitätsmedizin Berlin and lead author of the study, explained: "Our closed-loop perfusion system represents a paradigm shift in how advanced therapies can be delivered in vivo. By temporarily isolating an organ's circulation, we can achieve a level of precision and control that has not been possible with systemic delivery approaches."
Addressing Critical Delivery Barriers
Safe and efficient in vivo delivery remains one of the most significant barriers to the broad adoption of gene and cell therapies. Systemic administration of viral vectors, particularly adeno-associated viruses (搜索) (AAVs (搜索)), has been associated with dose-limiting toxicities, immune activation, and off-target organ exposure, limiting therapeutic index and commercial scalability.
The LRP platform directly addresses these limitations by enabling reversible and complete isolation of an organ's circulation. This approach allows therapeutic payloads to be delivered locally under tightly controlled physiologic conditions, potentially unlocking new therapeutic windows for modalities previously constrained by toxicity or delivery limitations.
Mayo Clinic Editorial Endorsement
In an accompanying editorial, Mayo Clinic experts described LRP as "a platform that closes the gap between promise and precision in gene delivery," highlighting its ability to overcome one of the most persistent limitations in advanced therapeutics: safe, organ-targeted delivery. The editorial characterized LRP as a "game-changer in the delivery of advanced therapies."
The Mayo Clinic experts noted that unlike historical isolated perfusion techniques, LRP is entirely endovascular and minimally invasive. This approach may enable scalable, outpatient-compatible precision delivery across multiple organs, including kidney, heart, liver, and lung, with broad applicability across gene therapy (搜索), RNA therapeutics, and regenerative medicine.
Broad Platform Potential
While the kidney served as the initial translational use case, the LRP platform is inherently organ-agnostic. Built on widely established interventional and perfusion principles, the technology is designed for adaptation across multiple solid organs, including the heart, lung, and liver, as well as for compartmentalized delivery to specific anatomical regions.
The platform demonstrates compatibility with a broad range of therapeutic modalities, including gene and cell therapies, gene-editing systems, RNA-based medicines (搜索), biologics (搜索), and high-potency pharmacologic agents. By enabling localized delivery with reduced systemic exposure, LRP has the potential to unlock new therapeutic windows for modalities previously constrained by toxicity or delivery limitations.
Strategic Spin-Out and Clinical Translation
To accelerate clinical translation and commercialization, DiNAQOR (搜索) has spun out this technology and its dedicated renal development programs into DiNATEQ AG (搜索), a newly established company headquartered in Switzerland. DiNATEQ AG will be led by Dr. Christian Thirion as Chief Executive Officer and Delegate of the Board of Directors.
Dr. Johannes Holzmeister, Founder, Chairman and CEO of the DiNAQOR (搜索) Group, commented: "This publication validates a delivery platform with implications far beyond a single organ or indication. Precision delivery is the critical enabler for the next generation of advanced therapies, and our closed-loop loco-regional perfusion technology establishes a new benchmark for safety, efficiency, and translational readiness."
Dr. Christian Thirion, Chief Executive Officer of DiNATEQ AG (搜索), added: "DiNATEQ has been established to translate this powerful delivery platform into meaningful clinical impact, starting with kidney gene therapy (搜索) and expanding beyond. The ability to precisely control exposure at the organ level changes the development equation for many advanced therapies."
Pharmaceutical Partnership Opportunities
For pharmaceutical and biotechnology partners, the LRP platform offers a differentiated delivery solution that can enhance the therapeutic index of advanced-therapy assets, enable lower effective doses, mitigate systemic toxicity (搜索) risk, and unlock programs previously constrained by delivery or safety limitations. The platform is designed to integrate seamlessly into existing clinical infrastructure, supporting efficient clinical translation and global scalability.
Dr. Josef el Andari, Co-Corresponding Author and Chief Scientific Officer at DiNAQOR (搜索), emphasized: "Independent validation from Mayo Clinic reinforces what we believe is a pivotal inflection point for advanced therapies. Too many transformative programs have failed not because the biology was wrong, but because delivery caused systemic toxicity (搜索). Loco-regional perfusion closes the gap between biological promise and clinical reality by enabling therapies to act precisely where they are needed."