Intellia R&D head says new gene-editing data shows path to human trials – Boston Business Journal

Posted: March 9, 2017 at 2:49 am

Broad Institute wins gene-editing patent case, boosting Editas shares Intellia moves into new HQ, says it plans to double headcount

John Leonard, executive vice president of R&D at Intellia

Cambridge-based Intellia Therapeutics had the biggest IPO of any local biotech in 2016, but shares of the company have lagged in recent months, and it has often been overshadowed by its gene-editing rival in Kendall Square, Editas Medicine.

On Wednesday, though, Intellia (Nasdaq: NTLA) wrested back the spotlight, announcing promising preclinical data from a study of its technology in mice that offers a pathway to potential trials involving humans.

John Leonard, executive vice president of R&D at Intellia

The company and partner Regeneron Pharmaceuticals (Nasdaq: REGN) have been developing a potential treatment for a rare hereditary disease called Transthyretin Amyloidosis, or ATTR. Intellias approach involves editing the defective genes that cause the buildup of the mutant protein responsible for the disease. The gene-editing tool, dubbed CRISPR/Cas9, is injected via lipid nanoparticles.

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Intellia R&D head says new gene-editing data shows path to human trials - Boston Business Journal

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