MilliporeSigma Awarded Australian CRISPR Nickase Patent for Foundational Genome-Editing Technology

• Patent covers paired Cas9 nickase technology to reduce off-target effects, advance gene therapy and research
• Expands company‘s foundational CRISPR cutting and integration IP necessary to correct genetic defects in gene therapy patients
• MilliporeSigma to license CRISPR-related patents to interested parties

Burlington, Massachusetts, August 15, 2018 — MilliporeSigma today announced that the Australian Patent Office has allowed the company’s patent application for the use of paired CRISPR nickases. Paired nickases represent a significant step in increasing safety by driving specificity through a highly flexible and efficient approach to reduce off-target effects. This improves CRISPR’s ability to fix diseased genes while not affecting healthy ones.

“We’ve made tremendous strides in recent years evolving CRISPR technology, and this is a pivotal time in scientific research,” said Udit Batra, CEO, MilliporeSigma. “MilliporeSigma’s paired nickase CRISPR technology is important for researchers who need highly accurate methods when developing treatments for difficult-to-treat diseases. This new patent allowance represents a significant advancement in safety for CRISPR-enabled therapeutics.”

The allowed patent application covers a foundational CRISPR strategy in which two CRISPR nickases are targeted to a common gene target and work together by nicking or cleaving opposite strands of a chromosomal sequence to create a double-stranded break. This process can optionally include an exogenous or donor sequence for insertion in the same manner as MilliporeSigma’s patented CRISPR integration technology. The requirement of two CRISPR binding events greatly reduces the chances of off-target cutting at other locations in the genome.

In addition to allowing a patent application on paired nickases, the Australian Patent Office recently announced the formal grant of MilliporeSigma’s 2017 CRISPR integration patent, following the withdrawal of four independent, anonymously filed oppositions.

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

Paired CRISPR nickase methods build on other technologies in MilliporeSigma’s CRISPR patent portfolio, including CRISPR integration. Commercial organizations need MilliporeSigma’s intellectual property for CRISPR-based insertion of DNA if they seek to correct genetic defects in the somatic cells of gene therapy patients. MilliporeSigma is licensing this patent portfolio for all fields of use.

As a company that for the past 14 years has been highly involved in genome-editing innovation, MilliporeSigma recognizes that genome editing has resulted in major advancements in biological research and medicine. At the same time, the growing potential of genome-editing technologies has opened scientific, legal and societal concerns. As both a user and supplier of genome-editing technology, MilliporeSigma supports research with genome editing under careful consideration of ethical and legal standards. MilliporeSigma’s parent company, Merck KGaA, Darmstadt, Germany, has established 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 taking into account scientific and societal issues to inform promising therapeutic approaches for use in research and applications.

MilliporeSigma was the first company to offer custom biomolecules for genome editing globally (TargeTron™ RNA-guided group II introns and CompoZr™ zinc finger nucleases), driving adoption of these techniques by researchers all over the world. MilliporeSigma was also the first company 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, MilliporeSigma supports development of gene- and cell-based therapeutics and manufactures viral vectors. In 2016, MilliporeSigma launched a genome-editing initiative aimed at advancing research in novel modalities — from genome editing to gene medicine manufacturing — through a dedicated team and enhanced resources, further solidifying the company’s commitment to the field.

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About the Life Science Business of Merck KGaA, Darmstadt, Germany

The Life Science business of Merck KGaA, Darmstadt, Germany, which operates as MilliporeSigma in the U.S. and Canada, has some 22,000 employees and 59 manufacturing sites worldwide, with a portfolio of more than 300,000 products focused on scientific discovery, biomanufacturing, and testing services.

Merck KGaA, Darmstadt, Germany, a leading science and technology company, operates across healthcare, life science, and electronics. Around 58,000 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 – the company is everywhere. In 2020, Merck KGaA, Darmstadt, Germany generated sales of € 17.5 billion in 66 countries.

The company holds the global rights to the name and trademark “Merck” internationally. The only exceptions are the United States and Canada, where the business sectors of Merck KGaA, Darmstadt, Germany operate as EMD Serono in healthcare, MilliporeSigma in life science, and EMD Electronics. Since its founding in 1668, scientific exploration and responsible entrepreneurship have been key to the company’s technological and scientific advances. To this day, the founding family remains the majority owner of the publicly listed company. For more information about Merck, KGaA, Darmstadt, Germany, visit  www.emdgroup.com.