Editas Medicine said today it will license from Massachusetts General Hospital (MGH) intellectual property and technology related to high-fidelity Cas9 nucleases and Cas9 protospacer adjacent motif (PAM) variants.
The value of the licensing agreement was not disclosed by Editas, which said the deal will enable it to address an expanded range of genetically defined diseases with potential for enhanced specificity.
None of the diseases were identified in the company’s announcement of the licensing agreement.
Editas is licensing technology developed by one of its scientific co-founders and consultants, J. Keith Joung, M.D., Ph.D. He also holds positions at MGH as associate chief for research, and as the Jim and Ann Orr MGH research scholar in the hospital’s Department of Pathology.
In a paper published January 28 in Nature, a research team led by Dr. Joung described their high-fidelity Cas9 variant—Streptococcus pyogenes Cas9-HF1—which is designed to reduce nonspecific DNA contacts. The researchers reported that SpCas9-HF1 rendered all or nearly all off-target events undetectable by genome-wide break capture and targeted sequencing methods.
The MGH team has also identified and characterized a series of novel S. pyogenes and Staphylococcus aureus Cas9 PAM variants that they said substantially increased the range of sites in the genome capable of being targeted for genome editing.
PAM is the region of the Cas9 protein that helps determine where Cas9 can bind to DNA. The PAM variants were detailed in the July 23, 2015 issue of Nature and the December 2015 issue of Nature Biotechnology.
“Keith Joung and his MGH team have done tremendous work in creating these novel, engineered forms of Cas9, and these advancements align fully with our highly differentiated genome editing platform. We are eager to deploy them and unlock their therapeutic potential,” Editas President and CEO Katrine Bosley said in a statement.
The collaboration comes less than a week after Editas partnered with the San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) to research and develop genome-edited hematopoietic stem cell (HSC) and T-cell therapies.