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

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Aug 15, 2018, 08:00 ET

- 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, Mass., Aug. 15, 2018 /PRNewswire/ -- 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.

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

"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 20,000 employees and 60 manufacturing sites worldwide, with a portfolio of more than 300,000 products enabling scientific discovery. Udit Batra is the global chief executive officer of MilliporeSigma.

Merck KGaA, Darmstadt, Germany completed its $17 billion acquisition of Sigma-Aldrich in November 2015, creating a leader in the $125 billion global life science industry.

Merck KGaA, Darmstadt, Germany is a leading company for innovative and top-quality high-tech products in healthcare, life science and performance materials. The company has five businesses – Biopharmaceuticals, Consumer Health, Allergopharma, Life Science and Performance Materials – and generated sales of €15.3 billion in 2017. Around 53,000 employees work in 66 countries to improve the quality of life for patients, to foster the success of customers and to help meet global challenges.

Merck KGaA, Darmstadt, Germany is the world's oldest pharmaceutical and chemical company – since 1668, the company has stood for innovation, business success and responsible entrepreneurship. Holding an approximately 70 percent interest, the founding family remains the majority owner of the company to this day. The company holds the global rights to the name and the trademark "Merck" internationally except for the United States and Canada, where the company operates as EMD Serono, MilliporeSigma and EMD Performance Materials.

SOURCE MilliporeSigma