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Duke Signs Exclusive Licensing Agreement with Leading Genome Editing Company
The agreement focuses on Gersbach’s work with genome engineering technologies known as CRISPR/Cas9 and TALENs.
CRISPR/Cas9 is an adaptive immune system found in bacteria that cuts out pieces of viral DNA and transplants them into the bacterium’s own DNA to defend against future re-infections. Not long ago, researchers discovered they could hijack this system to edit the sequence of the human genome. Similarly, the TALEN (transcription activator-like effector nuclease) technology uses proteins derived from bacteria to create new editing enzymes that can target and modify almost any gene sequence in any organism.
The agreement allows broad use of the technology developed in Gersbach’s lab for the prevention or treatment of human disease. To this point, Gersbach’s most notable work in that field is on Duchenne muscular dystrophy, a genetic disease affecting one in 3,500 newborn males that currently has no approved treatment and causes muscular deterioration, paralysis and eventual death, usually by age 25. Gersbach’s work is focused on using gene editing to correct the mutated gene that causes the disease, in contrast to treating the resulting symptoms of the disease. Gersbach has also pioneered the use of both CRISPR/Cas9 and TALEs for turning on genes in a way that could be used to treat degenerative disorders or compensate for genetic defects.
“Charlie’s deep expertise in both genome editing and in this area of biology is a tremendous asset as we begin to understand how to apply genome editing technologies to specific diseases,” said Katrine Bosley, chief executive officer of Editas Medicine. Gersbach is also serving as a scientific advisor to Editas.
Editas is a leading genome editing company and part of a transformational new area of healthcare—genomic medicine. The company was founded by the pioneers and world leaders in genome editing bringing specific expertise in CRISPR/Cas9 and TALENs technologies. The company’s mission is to translate its proprietary technology into novel solutions to treat a broad range of genetically-driven diseases.