Homology Medicines Acquires License to Novel AAV Technology to Develop Treatments for Central Nervous System Disorders

LEXINGTON, Mass., Sept. 15, 2016 /PRNewswire/ -- Homology Medicines, Inc., a genetic medicines company translating proprietary, next-generation gene editing and gene transfer technologies into novel treatments for patients, today announced that the Company has signed a license agreement with the California Institute of Technology for worldwide rights to a novel in vivo adeno-associated virus (AAV) technology.  Homology Medicines plans to combine Caltech's AAV technology with the Company's next-generation in vivo gene editing and gene transfer technology to develop treatments for central nervous system (CNS) disorders, with an initial focus on rare indications.

Under the terms of the agreement, Caltech granted Homology Medicines a co-exclusive license to specific patented AAV capsid sequences and peptides with demonstrated ability to cross the blood brain barrier, as well as a non-exclusive license to the patented methodology for in vivo screening, engineering and enrichment of novel AAV capsids.

"With this agreement we bring together our proprietary AAVs and in vivo gene editing technologies with novel AAV capsid sequences and peptides that facilitate and enhance blood brain barrier transport," said Arthur Tzianabos, Ph.D., Chief Executive Officer of Homology Medicines. "The combination of these technologies and Homology Medicines' expertise in developing and commercializing rare disease therapies provides us with a unique ability to target specific cell types in the CNS and the potential to cure a wide range of diseases."

The technology underlying the agreement is based on the work of Dr. Benjamin Deverman, Dr. Viviana Gradinaru, and the late Dr. Paul Patterson at Caltech, and was featured in a Nature Biotechnology publication earlier this year (Deverman, et al. Nat Biotechnol 2016).  The research has generated and enabled the in vivo discovery of new AAV variants that efficiently and widely transduce the adult mouse brain following intravenous injection.  In these studies, transduction, the process by which foreign DNA is introduced into a cell via virus or viral vector, was at least 40-fold more efficient in the CNS than that of AAV9, the current standard serotype that can cross the blood brain barrier.  Caltech's technology transduces neuronal cell types such as astrocytes, glial cells and neurons in the CNS, across multiple regions of the mouse brain at significantly higher efficiencies.  Homology Medicines shares this co-exclusive license from Caltech with Voyager Therapeutics.

Homology Medicines' platform technology is based on a newly discovered family of AAVs that can edit genes in vivo by harnessing a naturally-occurring and precise DNA repair mechanism known as homologous recombination.  Homology Medicines' approach, called AMEnDR™ (AAV-Mediated Editing by Directed Homologous Recombination) technology, can correct and edit a gene mutation through in vivo delivery of a single AAV vector.  Importantly, AMEnDR™ does not use exogenous nucleases that are required for multi-component platforms, such as CRISPR, which carry the risk of inducing off-target effects.

About Homology Medicines, Inc.
Homology Medicines is a genetic medicines company translating proprietary, next generation gene editing and gene therapy technologies into novel treatments for patients with rare diseases. The combination of a management team that has successfully developed and commercialized rare disease therapies, and a new multidimensional technology platform uniquely positions the Company to move beyond the current limitations of gene therapy and editing approaches to improve patient care. Homology Medicines has built foundational intellectual property on gene editing and gene therapy using vectors derived from naturally occurring human adeno-associated viruses (AAVs). The Company's technology is precise, on-target and highly efficient for in vivo editing of genetic mutations. The unique team and technology create a significant opportunity for Homology Medicines to rapidly advance a diverse pipeline of new medicines that address and potentially cure the underlying cause of genetic diseases. For more information, please visit www.homologymedicines.com.

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SOURCE Homology Medicines, Inc.

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