Hunterian Medicine® Licenses Inscripta’s MAD7™ Nuclease to Advance Gene Editing Research and Development

Inscripta and Hunterian pioneer new program to provide access to novel CRISPR enzymes for biotherapeutic development

Inscripta, Inc., the digital genome engineering company, today announced that it has granted a non-exclusive license to Hunterian Medicine^®, a gene-editing and gene therapy company, for access to the MAD7™ nuclease, one of several CRISPR nucleases in Inscripta’s MADzymes™ family of enzymes. Inscripta has introduced a new commercial licensing model for biopharma companies that further democratizes access to CRISPR-based gene-editing.

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Under the terms of its license, Hunterian will have the right to use the MAD7 nuclease or improved MADzyme nucleases in its gene-editing programs to develop human therapeutics. This complements Hunterian’s proprietary technology that enables in vivo delivery of CRISPR via a single adeno-associated virus (AAV) vector.

Hunterian is the first biopharmaceutical organization to license the MAD7 nuclease under Inscripta’s commercial licensing program. Inscripta originally released the MAD7 nuclease free for academic and commercial R&D in 2017. Today’s announcement represents an extension of Inscripta’s licensing program to expand access to CRISPR and address limitations in CRISPR-based therapeutic development and commercialization.

"The vast therapeutic potential of gene-editing has one primary barrier: Delivery. Hunterian solved this problem by enabling CRISPR systems to fit inside a single AAV, the gold standard for gene delivery,” said Dr. Vinny Jaskula-Ranga, President and CEO of Hunterian Medicine. “Inscripta is similarly reducing barriers to innovation by providing access to its MAD7 nuclease and other improved MADzyme nucleases. For therapeutic indications, MAD7 is a particularly important alternative to commonly used Cas9 nucleases given that it has significantly fewer off-target effects."

"Gene-editing has demonstrated vast potential for breakthrough innovation in multiple industries. We see incredible opportunity and potential for CRISPR in therapeutic applications; however, we constantly hear biopharma companies talk about limitations in their access,” said Sri Kosaraju, President and CEO of Inscripta. “By providing more flexible access to MADzyme nucleases to companies such as Hunterian, Inscripta believes that we can help drive scientific progress and expand the use of CRISPR across more application areas and industries in the bioeconomy. We look forward to working with Hunterian to enable their AAV-based gene-editing programs to address significant unmet medical needs.”

About MADzymes

Inscripta developed the Madagascar family of nucleases (“MADzymes™”) and released the MAD7 nuclease in December 2017 to both commercial and academic researchers — without licensing fees or reach-through royalties for most applications — to improve access to CRISPR. Royalty-bearing commercial licenses apply only in a few scenarios where the MAD7 nuclease is used on a continuous basis for manufacturing or if a product physically contains the MAD7 nuclease. Since 2017, the MAD7 nuclease has been adopted widely, and multiple publications detail the use of MAD7 in microbes, plants, mammalian cell lines, stem cells, and animal models. Inscripta continues to innovate and find new and improved MADzyme nucleases, including higher-fidelity MAD7 nuclease variants, and is licensing the MAD7 nuclease commercially, including licensing for therapeutic uses in gene and cell therapy. For more information about licensing MADzymes, please visit inscripta.com/mad7.

About Inscripta

Inscripta is a life science technology company enabling scientists to solve some of today’s most pressing challenges with the first benchtop system for genome editing. The company’s automated Onyx™ platform, consisting of an instrument, consumables, assays, and software, makes CRISPR-based genome engineering of microbes accessible to any research lab. Inscripta supports its customers around the world from facilities in Boulder, Colo.; San Diego and Pleasanton, Calif.; and Copenhagen, Denmark. To learn more, visit Inscripta.com and follow @InscriptaInc.

About Hunterian Medicine^®

Hunterian Medicine, headquartered in Cambridge, Massachusetts with a laboratory in the LifeBridge Health BioIncubator in Baltimore, Maryland, is a pre-clinical gene-editing and gene therapy company working to cure genetic diseases using its innovative in vivo delivery technology, which can deliver the commonly used SpCas9, high-fidelity variants, Cas12a, PAM variants and many other systems through a single adeno-associated virus (AAV). The company’s platform technology provides a solution to the AAV gene delivery problem for key areas of (1) CRISPR gene-editing and (2) gene therapy applications. The company is developing a pipeline of gene-editing and gene therapy therapeutics to address diseases of significant unmet need. For more information, please visit www.hunterian.com.

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