CRISPR-THERAPEUTICS

CRISPR Therapeutics, a biopharmaceutical company focused on the breakthrough gene-editing technology CRISPR-Cas9, has raised $25 million in a series A investment from Versant Ventures. The company today also announced a founding team comprising high-profile experts in diverse fields of science including CRISPR-Cas9, genome editing, stem cell biology, advanced drug delivery technologies, RNA interference and gene silencing.

The funding and the team’s collective expertise will allow CRISPR Therapeutics to translate its technology into important new gene medicines that have the potential to cure serious genetic diseases at the molecular level.

Cas9 is an enzyme that can be easily programmed with RNA to cut DNA at targeted sites within the genome,^1-2 enabling the deletion, insertion or correction of target genes with surgical precision. It allows scientists and clinicians to mutate genes of interest and to correct specific target genes, to tackle both recessive and dominant genetic diseases. CRISPR-Cas9 genome editing offers significant advantages over traditional gene therapy approaches, which to date have only been useful in correcting some recessive genetic disorders.

“The efficiency and ease of use of CRISPR-Cas9 have led to the scientific community rapidly adopting the technology on an unprecedented scale,” said Dr Rodger Novak, Chief Executive Officer of CRISPR Therapeutics. “We look forward to leveraging this potentially game-changing technology to provide new gene medicines to patients suffering from serious diseases.”

Dr Tom Woiwode, Venture Partner of Versant Ventures, said: “We believe that CRISPR Therapeutics has immense potential, as it is uniquely positioned to translate this technology into human therapeutics, thanks to its foundational intellectual property and its multi-disciplinary team of experts, including senior industry executives, world-renowned academics, and clinicians.”

CRISPR Therapeutics’ scientific founders are:

• Dr Daniel Anderson, the Sam Goldblith Associate Professor at the Massachusetts Institute of Technology and an intramural member of the Koch Institute for Integrative Cancer Research
• Dr Emmanuelle Charpentier, a professor at the Helmholtz Centre for Infection Research and Hannover Medical School, Germany and the Laboratory for Molecular Infection Medicine at Umeå University, Sweden, who holds an Alexander von Humboldt professorship for research in Germany
• Dr Chad Cowan, an associate professor at Harvard University in the Department of Stem Cell and Regenerative Biology, and at Massachusetts General Hospital, with appointments in the Center for Regenerative Medicine, the Cardiovascular Research Center and the Center for Human Genetics Research
• Dr Craig Mello, a Nobel prize-winning investigator at the Howard Hughes Medical Institute, the Blais University Chair in Molecular Medicine, and co-director of the RNA Therapeutics Institute at the University of Massachusetts Medical School
• Dr Matthew Porteus, an associate professor of pediatrics, Department of Pediatrics; Divisions of Hematology/Oncology and Human Gene Therapy, at Stanford School of Medicine

CRISPR Therapeutics has secured rights to the foundational intellectual property covering the use of CRISPR-Cas9 genome editing in human therapeutics. The company in-licensed the technology from Dr Charpentier, who co-invented the CRISPR-Cas9 technology.^1-2

“CRISPR-Cas9 has truly democratized genome editing, with many areas of research being transformed due to the technology’s ease of use and broad applicability,” said Dr Charpentier. “The collective efforts of the broader academic research community are in turn driving tremendous progress in the field.”

About CRISPR Therapeutics

CRISPR Therapeutics is a biopharmaceutical company created to translate CRISPR-Cas9, a breakthrough gene-editing technology, into transformative medicines for serious human genetic diseases. Its vision is to cure serious human diseases at the molecular level using CRISPR-Cas9. CRISPR Therapeutics is a Swiss company with operations in London, UK. www.crisprtx.com

About Versant Ventures

Versant Ventures is a leading venture capital firm that specializes in investments in game-changing medical devices, biopharmaceuticals, and other life science opportunities. Founded in 1999, the firm consists of an experienced team of managing directors committed to helping entrepreneurs build successful companies that impact healthcare and improve quality of life. Versant has offices in the US, Canada and Switzerland. www.versantventures.com

References

1. Deltcheva, E et al. (2011). CRISPR RNA maturation by trans-encoded small RNA and host factor RNase III. Nature, (471), 602-607.

2. Jinek, M and Chylinski, K et al. (2012). A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science, (337), 816-821.

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Email: pete.chan@tudor-reilly.com

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