-- Patent covers paired Cas9 nickase technology to advance gene therapy and 
research, reduce off-target effects

-- Marks company's 13th CRISPR patent overall

-- Actively licensing CRISPR-related patents to interested parties

Merck ( 
https://c212.net/c/link/?t=0&l=en&o=2388064-2&h=3840555483&u=https%3A%2F%2Fc212.net%2Fc%2Flink%2F%3Ft%3D0%26l%3Den%26o%3D2388064-2%26h%3D328798815%26u%3Dhttps%253A%252F%252Fwww.merckgroup.com%252F%26a%3DMerck&a=Merck 
), a leading science and technology company and leader in genome editing, today 
announced that the Canadian Patent Office has allowed Merck's patent 
application directed to the use of paired CRISPR nickases in eukaryotic cells. 
The patent provides an important and more specific solution for scientists who 
need accurate methods when developing treatments for difficult-to-treat 
diseases that ultimately reach the patient.

Photo - https://mma.prnewswire.com/media/827881/CRISPR_EN_Merck.jpg 

"This patent allowance marks another advancement in safety for CRISPR-enabled 
therapeutics, as it covers technology that improves CRISPR's ability to fix 
diseased genes while not affecting healthy ones," said Udit Batra, member of 
the Merck Executive Board and CEO, Life Science. "Merck has been at the 
forefront of genome-editing innovation for 15 years and this allowance further 
expands our foundational CRISPR cutting and integration intellectual property 
to help scientists advance gene therapy research."

CRISPR technology is a core competency for Merck. The company supports research 
with genome editing under careful consideration of ethical and legal standards. 
Merck has established an independent, external Bioethics Advisory Panel ( 
https://c212.net/c/link/?t=0&l=en&o=2388064-2&h=3407059452&u=https%3A%2F%2Fc212.net%2Fc%2Flink%2F%3Ft%3D0%26l%3Den%26o%3D2388064-2%26h%3D3156828697%26u%3Dhttp%253A%252F%252Freports.merckgroup.com%252F2017%252Fcr-report%252Fproducts%252Fbioethics.html%26a%3DBioethics%2BAdvisory%2BPanel&a=Bioethics+Advisory+Panel 
) to provide guidance for research in which its businesses are involved, 
including research on or using genome editing, and has defined a clear 
operational position ( 
https://c212.net/c/link/?t=0&l=en&o=2388064-2&h=3114576659&u=https%3A%2F%2Fc212.net%2Fc%2Flink%2F%3Ft%3D0%26l%3Den%26o%3D2388064-2%26h%3D3208219101%26u%3Dhttps%253A%252F%252Fwww.merckgroup.com%252Fcontent%252Fdam%252Fweb%252Fcorporate%252Fnon-images%252Fcompany%252Fresponsibility%252Fen%252Fregulations-and-guidelines%252Fgenome-editing-principle-EN.pdf%26a%3Dposition&a=position 
) taking into account scientific and societal issues to inform promising 
therapeutic approaches for use in research and applications.

This Canadian patent marks Merck's second in Canada and the 13th worldwide. 
Similar patents were granted in Australia and Europe in late 2018.  This latest 
patent covers paired nickases, which drive specificity through a highly 
flexible and efficient approach to reduce off-target effects.

Paired nickases are two CRISPR nickases targeted to a common gene target that 
work together by nicking or cleaving opposite strands of a chromosomal sequence 
to create a double-stranded break. This process can include an exogenous or 
donor sequence for insertion in the same manner as Merck's CRISPR integration 
technology. The requirement of two CRISPR binding events greatly reduces the 
chances of off-target cutting at other locations in the genome.

Merck's CRISPR integration patent portfolio includes granted patents in 
Australia, Canada, China, Europe, Israel, Singapore and South Korea. These 
CRISPR patents are for chromosomal integration or cutting of the sequence of 
eukaryotic cells and insertion of a DNA sequence, to make a desired genomic 
change.

Merck is licensing its entire patent portfolio for all fields of use.

Merck was the first company to offer custom biomolecules for genome editing 
globally (TargeTron (TM) RNA-guided group II introns and CompoZr(TM) zinc 
finger nucleases), driving adoption of these techniques by researchers all over 
the world. Merck was also the first to manufacture arrayed CRISPR libraries 
covering the entire human genome, accelerating disease cures by allowing 
scientists to explore more questions about root causes.

In addition to basic genome-editing research, Merck supports development of 
gene- and cell-based therapeutics and manufactures viral vectors. The Life 
Science business of Merck has a dedicated genome-editing group aimed at 
advancing research in novel modalities — from genome editing to gene medicine 
manufacturing — further solidifying the company's commitment to the field.

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About Merck

Merck, a leading science and technology company, operates across healthcare, 
life science and performance materials. Around 51,000 employees work to make a 
positive difference to millions of people's lives every day by creating more 
joyful and sustainable ways to live. From advancing gene- editing technologies 
and discovering unique ways to treat the most challenging diseases to enabling 
the intelligence of devices – Merck is everywhere. In 2017, Merck generated 
sales of (EUR)15.3 billion in 66 countries.

Scientific exploration and responsible entrepreneurship have been key to 
Merck's technological and scientific advances. This is how Merck has thrived 
since its founding in 1668. The founding family remains the majority owner of 
the publicly listed company. Merck holds the global rights to the "Merck" name 
and brand. The only exceptions are the United States and Canada, where the 
business sectors operate as EMD Serono in healthcare, MilliporeSigma in life 
science, and EMD Performance Materials.

SOURCE: Merck

CONTACT: karen.tiano@emdmillipore.com, +49 6151 72 44461