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Genome-Editing Company Raises $94 Million in IPO

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3 February 2016. Editas Medicine, developer of treatments for disease that harness editing of the human genome, is raising $94.4 million in its initial public stock offering. The company, trading on the Nasdaq exchange under the symbol EDIT, issued 5.9 million shares priced at $16.00. As of 12 noon on 3 February, the stock is trading at $17.00 a share, up 6.25 percent, while the Nasdaq overall is down 0.8 percent.

Founded in 2013, Editas Medicine is designing therapies with the ability to turn off and on and repair genes causing disease. The company’s technology uses a technique called clustered, regularly interspaced short palindromic repeats, or Crispr, and related Crispr-associated protein 9, together known as CRISPR-Cas9. With CRISPR-Cas9, the Cas9 protein binds to targeted RNA molecules generated by the human genome. The RNA molecules then guide Cas9 proteins to specific genes needing repair, making it possible to address root causes of many diseases.

In December 2014, the Cambridge, Massachusetts company licensed the work of its founders George Church and David Liu of Harvard University and Feng Zhang of the Broad Institute, a medical research center at Harvard and MIT. Church, Liu, and Zhang are pioneers in the development of Crispr-Cas9 and serve as scientific advisers to Editas. Since September 2015, as reported in Science & Enterprise, Zhang published studies demonstrating methods to simplify the Crispr technique and improving its accuracy.

Researchers from Editas in March 2015 demonstrated repair of genetic defects causing sickle-cell disease, an inherited blood disorder affecting hemoglobin that delivers oxygen to cells in the body. The team applied Crispr/Cas9 to a process known as gene conversion, where the mutated gene is repaired with a different, but closely related gene.  That demonstration used lab cultures and was not tested in humans or lab animals.

An international summit on genome editing in December 2015 established voluntary guidelines for further research and development of Crispr and related techniques. Those guidelines allow continued work with editing of genes in somatic cells, where the genomes are not transmitted to future generations. Examples include editing genes for sickle-cell disease or improving the ability of immune cells to target cancer. The guidelines, however, ask that clinical applications to germ line cells — for example, in human embryos — not proceed until much more is known about their medical and social consequences.

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