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Genome Editing Start-Up Secures $25 Million in First Round

Emmanuelle Charpentier

Emmanuelle Charpentier (Helmholtz Centre for Infection Research)

24 April 2014. Biopharmaceutical start-up company CRISPR Therapeutics in Basel, Switzerland raised $25 million in its first funding round. The company develops engineered gene therapies with a technology developed by one of its scientific founders, and is backed in this round by Versant Ventures, a venture capital company in Menlo Park, California.

CRISPR Therapeutics’ technology is based on the research of Emmanuelle Charpentier, a professor at the Helmholtz Centre for Infection Research in Braunschweig, Germany. Charpentier is also affiliated with the Hannover Medical School also in Germany and the molecular biology department at Umea University in Sweden. Her research discovered the capability of CRISPR — short for clustered regularly interspaced short palindromic repeats — to alter human genomes with an enzyme known as Cas9.

The Cas9 enzyme — Cas is short for CRISPR-associated — can program RNA to silence genes and provide immunity against invading genetic material. Cas9 also harnesses RNA to cut DNA at precise points in genomes, making it possible to delete, insert, or correct defects in human genomes. Charpentier led research teams that published their findings in the journal Science in 2012, and an article in Nature a year earlier.

CRISPR Therapeutics plans to develop treatments based on CRISPR-Cas9 editing for genetic disorders, including those where an abnormal gene from one parent can cause disease, called dominant inheritance. Genomic editing techniques up to now were applied only to genetic conditions where mutations are inherited from both parents, or recessive inheritance. Huntington’s disease is an example of a dominant inheritance disease; cystic fibrosis is a recessive inheritance disorder.

“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,” says Charpentier in a company statement.”The collective efforts of the broader academic research community are in turn driving tremendous progress in the field.”

Charpentier is one of five scientific founders of CRISPR Therapeutics. The other founders are:

Craig Mello, a 2006 Nobel laureate and co-director of the RNA Therapeutics Institute at the University of Massachusetts Medical School

Daniel Anderson of the Koch Institute for Integrative Cancer Research at MIT

Chad Cowan of the Department of Stem Cell and Regenerative Biology at Harvard University

Matthew Porteus, a professor of pediatrics and researcher in gene therapy at the Stanford University School of Medicine

Versant Ventures, that’s providing early financing for CRISPR Therapeutics, specializes in investments in life science enterprises, focusing on medical devices and biopharmaceuticals, with $1.6 billion currently under management, according to the company. Bradley Bolzon, managing director of Versant Ventures, chairs the CRISPR Therapeutics board.

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