CMRI expands gene therapy research partnership with LogicBio Therapeutics

Children’s Medical Research Institute (CMRI) is pleased to announce that its partnership with LogicBio Therapeutics to develop the next generation of viral vectors for gene therapy applications has been extended for another two years and two new target tissues.

In 2018, LogicBio Therapeutics, Lexington MA, USA, a clinical stage genetic medicines company, entered into a collaboration with Associate Professor Leszek Lisowski, Ph.D., MBA (Head of the Translational Vectorology Research Unit at CMRI) and Professor Ian Alexander, MBBS, Ph.D. (Head of the Gene Therapy Research Unit at CMRI and Sydney Children’s Hospitals Network) to develop next-generation bioengineered liver-directed adeno-associated viral (AAV) vectors that overcome many of the functional limitations of the AAV vectors in clinical studies today.

The AAV Development Program at CMRI is led by Dr Marti Cabanes-Creus, a global expert in AAV vector biology and vector bioengineering. Dr Cabanes-Creus is originally from Barcelona, Spain, and received his PhD from University College London, UK.

As part of the efforts to develop new and improved vectors compatible with clinical applications, the CMRI team apply a range of advanced genetic and molecular biology techniques ranging from methods based on the 2018 Chemistry Nobel Prize-awarded concept of Directed Evolution to bioinformatics techniques utilizing advanced computational methods, such as machine learning.

Building on the critical insights gained into the mechanisms of AAV vector interaction with primary human cells, and access to proprietary novel highly functional AAV vectors developed during the initial two years of the partnership, the team is now combining advanced computational strategies with access to primary patient cells to develop the next generation of AAV vectors for clinical implementation. Importantly, the tools and strategies developed are not limited to human liver and can be used to develop improved AAV vectors for therapeutic delivery to other human tissues, which enables expansion of the AAV Development Program into other clinically important tissues.

“The outcomes of the project to date have been very exciting,’’ A/Prof. Lisowski said. “The work led to the development of a set of highly functional vectors which we are confident will revolutionize clinical gene therapy applications and will put many more challenging indications within the technological reach, bringing hope to patients and their families. In addition, the collaboration with LogicBio Therapeutics led to three patent applications and two publications in prestigious scientific journals.”

“Because of these promising results, while we extend and expand the AAV Development Program, we are also taking it to the next level. We have learned a lot about which elements of the vector capsid (the outer shell) are critical to its function and now we can take a more targeted approach to get the best results.” Dr Cabanes-Creus said. “We understand better how the viral vectors interact with human cells, what makes the interaction stronger and what weakens it. We are now applying machine learning and artificial intelligence approaches to the best vectors developed during the last two years to further improve their function and manufacturability, and to minimize reactivity with the human immune system.”

“This collaboration highlights how important it is for academic and commercial teams to interact and collaborate to achieve the greatest success” Prof. Alexander added. “We must not compete with each other but rather find a way to work together to increase the speed and efficiency with which new therapies are being developed and delivered to patients”.

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