DiNAQOR, a genetic medicine platform company focused on addressing severe inherited cardiac diseases, today announced it has entered into a research collaboration with Children’s Medical Research Institute (CMRI) in Australia to develop novel bioengineered adeno-associated virus (AAV) capsids to route gene therapy directly to human cardiac muscle.
| ZURICH-SCHLIEREN, Switzerland and WESTMEAD, NSW, Australia, Jan. 10, 2022 /PRNewswire/ -- DiNAQOR, a genetic medicine platform company focused on addressing severe inherited cardiac diseases, today announced it has entered into a research collaboration with Children’s Medical Research Institute (CMRI) in Australia to develop novel bioengineered adeno-associated virus (AAV) capsids to route gene therapy directly to human cardiac muscle.
Under the terms of the agreement, DiNAQOR has the option to obtain an exclusive license for capsids co-developed with CMRI for both cardiovascular and kidney diseases. As part of the collaboration, CMRI will make available its extensive library of AAV capsids, which are the protein shells surrounding viruses that act as a delivery mechanism for gene therapies. DiNAQOR will provide access to its proprietary engineered heart tissue (EHT) technology and animal tissue to enable the most effective and clinically-impactful screen of the CMRI capsids to identify novel cardiac-specific capsid variants. “We are honored to be working closely with CMRI, a pioneer in the field of gene therapy and a world leader in the design of capsids to deliver these medicines,” said Eduard Ayuso, D.V.M., Ph.D., Chief Technology Officer at DiNAQOR. “Our aim is to develop new capsids that can target the heart more efficiently at lower doses.” DiNAQOR will screen capsids for transduction systemically and tailor AAV capsids for loco-regional perfusion (LRP) administration. DiNAQOR’s LRP system enables gene therapies to be routed directly to the cardiac muscle, maximizing biodistribution and transduction of the cardiac cells. This new approach, which is actively being used in several pre-clinical studies, may minimize potential adverse effects of systemic gene therapy delivery while lowering the cost. “This will be an exciting collaboration, and it is consistent with our strategy to align with world leading academic institutions to expand our R&D efforts, platform capabilities and our pipeline,” said Johannes Holzmeister, M.D., Chairman and CEO of DiNAQOR. “CMRI has a stellar team, and we look forward to working closely with them to make a real difference in the lives of patients.” The CMRI team is led by Associate Professor Leszek Lisowski, Ph.D., MBA, an expert in viral vector-based gene therapies, vectorology and genotoxicity. “We look forward to working with the team at DiNAQOR to identify novel capsids that may improve the standard of care for patients with heart disease,” commented Associate Professor Lisowski. “We are optimistic that novel capsids, administered with DiNAQOR’s LRP system, will form a foundation of novel advanced therapies to benefit millions of affected patients world-wide.” About Children’s Medical Research Institute About DiNAQOR Contacts: For Children’s Medical Research Institute:
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