Editas Medicine (EDIT) said Wednesday its CRISPR-based treatment led to meaningful improvements for one patient with a blindness-causing genetic disorder, but EDIT stock tumbled.
This was the first data on Editas’ treatment, known as EDIT-101. The company tested a midsize dose of EDIT-101 in four patients with Leber congenital amaurosis 10. One patient showed improvements in functional vision through six months. Another patient was considered “stable” through three months.
But the results fell short on one measure of an experimental treatment from ProQR Therapeutics. The company is working on an RNA-based treatment for the same blindness-causing disorder. Patients who received ProQR’s drug showed a stronger improvement on a measure of “best corrected visual acuity.”
“We do expect (Editas) stock to get some pressure at the open,” RBC Capital Markets analyst Luca Issi said in a report.
In morning trading on today’s stock market, EDIT stock plummeted 16.7%, near 43.70. The move pushed shares below their 200-day line for the first time since early August, according to MarketSmith.com.
EDIT Stock Dives On Gene-Editing Test
Editas is using a method called CRISPR to edit genes inside the body. Most CRISPR companies are focusing first on editing cells outside the body before infusing them into patients.
Early data suggest the drug is doing its job, editing genes inside the body, Editas said in a news release. Evidence includes improvements in best corrected visual acuity, light sensitivity and ability to navigate a standard navigation course. One patient showed improvements through six months. Another was stable at three months.
“These encouraging results provide a proof of concept on our in vivo gene-editing platform and increase Editas’ confidence in the broad potential of our gene-editing technology to address additional serious disease,” Editas Chief Medical Officer Lisa Michaels said in a written statement.
Most side effects were the result of the injection in the eye, Editas said.
But EDIT stock bears could note there was some inflammation in a part of the eye. Patients received oral steroids to control the inflammation. Editas is also testing a high dose in another group of adults, and a midsize dose in children with the disease.
Follow Allison Gatlin on Twitter at @IBD_AGatlin.
YOU MAY ALSO LIKE:
Need Your Help Today. Your $1 can change life.