CAMBRIDGE, Mass., Nov. 15, 2018 (GLOBE NEWSWIRE) -- LogicBio Therapeutics, Inc. (LOGC), a genome editing company focused on developing medicines to durably treat rare diseases, today announced the launch of a partnership to develop new viral vectors with Children’s Medical Research Institute (CMRI) of Australia, a world leader in the areas of gene therapy, childhood cancer, embryology and neurological diseases.
The newly formed AAV Development Program will focus on developing next-generation synthetic adeno-associated virus (AAV) capsids capable of overcoming many of the current limitations of existing viral vectors. LogicBio will hold exclusive worldwide commercial rights to vectors developed under the partnership, with the goal of commercializing the new capsids as widely as possible.
"We’re optimistic that we can significantly improve the performance of AAV vectors through this collaboration,” said Fred Chereau, CEO of LogicBio. “These next-generation vectors will strengthen our platform and expand our pipeline. We also expect them to bolster other important genetic medicine approaches to provide benefits to more patients.”
The AAV Development Program will be led by Professor Ian Alexander, MBBS, Ph.D., Head of the Gene Therapy Research Unit at CMRI and Sydney Children’s Hospitals Network (SCHN), and Dr. Leszek Lisowski, Ph.D., MBA, leader of the Translational Vectorology Group at CMRI.
Dr. Lisowski, a scientific co-founder of LogicBio Therapeutics, is well-known for his work in the Stanford University lab of Professor Mark A. Kay, M.D., Ph.D. which led to the development of the novel synthetic liver-tropic capsid AAV-LK03. This capsid, which was functionally characterized in a partnership between Stanford and CMRI, together with SCHN, is now being used in the clinic in various gene therapy programs. AAV-LK03 is also being evaluated by LogicBio to deliver corrective transgenes to pediatric patients, using GeneRide™, LogicBio’s proprietary genome editing technology.
Over the past few decades, AAV has emerged as a potent, versatile and safe platform for gene delivery into cells in vitro and in vivo. Current clinical applications of gene therapy and gene editing are largely based on families of AAVs discovered over 10 years ago. There is now a pressing need to identify new and improved vectors with greater tissue tropism, improved immunogenic profile, enhanced manufacturability and optimization for delivering different payloads.
“We are confident that we can further improve on the performance of current AAV vectors, expanding their utility in a range of tissues, while also improving manufacturability and reducing cost,” said Dr. Lisowski.
CMRI and its clinical partner, SCHN, have long contributed to pioneering work in the clinical translation of cell and gene therapies, including being the first in Australia to treat a genetic disease (SCID-X1) by gene therapy.
"With the AAV Development Program, CMRI continues to build upon its groundbreaking work to facilitate the development of novel vectors and therapies, as well as the clinical translation of those efforts. Our goal is to ensure that these life-saving therapies are made available to our patients and the wider community," said Professor Alexander.
About LogicBio Therapeutics
LogicBio Therapeutics is a genome editing company focused on developing medicines to durably treat rare diseases in patients with significant unmet medical needs using GeneRide™, its proprietary technology platform. GeneRide™ enables the site-specific integration of a therapeutic transgene in a nuclease-free and promoterless approach by relying on the native process of homologous recombination to drive lifelong expression. Headquartered in Cambridge, Mass., LogicBio is committed to developing medicines that will transform the lives of pediatric patients and their families.
For more information, please visit www.logicbio.com.
About Children’s Medical Research Institute (CMRI)
CMRI pioneered microsurgery, immunizations against lethal childhood illnesses and care for premature babies, improving the lives of countless Australian children over the last 60 years. Today, CMRI is an independent institute and the site of world-leading research in the areas of cancer, neurobiology, embryology, proteogenomics and gene therapy.
CMRI collaborates with scientists all over the world to move research forward. It also provides important resources for scientists in Australia. It operates CellBank Australia™, the only national repository of cell cultures in Australia, major biomedical proteomics facilities, as well as advanced gene therapy and gene engineering laboratories creating treatments of the future. In addition, CMRI houses the ACRF Cancer Centre, which includes the ACRF Centre for Kinomics, ACRF Telomere Analysis Centre and ProCan®, whose combined efforts are to understand, improve diagnosis of, and discover new treatments for all types of cancer.
CMRI, based in Western Sydney, is an affiliate of the University of Sydney and a founding member of the Westmead Research Hub, Sydney Health Partners and of Paediatrio, all collaborations aimed at improvements in science and translation of discoveries into health treatments.
CMRI’s achievements are made possible by a network of devoted community supporters, as well as the iconic Jeans for Genes® fundraising campaign. www.cmri.org.au
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