The Editas IPO was priced at $18. It held up well before our note of caution came out before settling at about $13 a few days later. From there it vaulted up past $30 as attention on the name increased - even HBO did a special on gene-editing - perfect storm.
Editas is developing a proprietary genome editing platform based on CRISPR/Cas9 technology. CRISPR/Cas9 uses a protein-RNA complex composed of the Cas9 enzyme bound to a guide RNA molecule designed to recognize a particular DNA sequence. The RNA molecule guides the Cas9 complex to the location in the genome that requires repair. Once there, the complex makes a specific cut in the DNA, ultimately triggering the cell's DNA repair machinery to address the genetic defect. Our platform consists of four interrelated components: nuclease engineering, delivery, control and specificity, and directed editing. These components are designed to develop medicines that specifically address a wide variety of genetic targets, reach the site of disease safely and effectively, tightly and specifically control the editing process, and drive the right kind of genetic repair. This drug discovery platform uses the flexibility of CRISPR/Cas9 technology to enable rapid reprogramming of the Cas9-guide RNA complex with the potential to direct it to almost any site in the human genome. Using this platform, Editas plans to develop and advance a broad range of therapies for genetically defined diseases. Our product development strategy is to target genetically defined diseases with an initial focus on debilitating illnesses where there are no approved treatments and where the genetic basis of disease is well understood. Their most advanced research program is designed to address Leber Congenital Amaurosis type 10, or LCA10, a specific genetic form of progressive blindness with no available therapies or potential treatments in clinical trials in either the United States or European Union. The localization of LCA10 disease in the eye allows for application of the technology in a context that is confined and relatively uncomplicated compared to systemic illnesses. Combinations of Cas9 and guide RNA pairs have been tested in cells that were taken from patients with a specific mutation that causes LCA10 and demonstrated restoration of normal messenger RNA and protein expression, suggesting that these can correct the LCA10 gene defect in these cells. An IND is expected in 2016 which will lead to clinical trials in 2017. Success of this proof-of-concept in a disease of the eye has the potential to validate the use of this techology in applications to other organs and diseases.