Investors unsure on touted promise with CRISPR therapy

Intellia Therapeutics (Nasdaq: NTLA) and Regeneron Pharmaceuticals (Nasdaq: REGN) presented positive interim results from a well-watched Phase I trial early Friday.

The ongoing trial is studying NTLA-2001, an investigational, in vivo CRISPR/Cas9 genome editing therapy in development as a single-dose treatment for transthyretin (ATTR) amyloidosis.

"We look forward to completing the Phase I study as we advance closer to a potential pivotal trial, which we expect will include patients in the USA"With the data appearing to display the potential of NTLA-2001 to halt and even reverse the underlying cause of the rare disease, and the wider power of CRISPR-based technologies, shares in Intellia moved upwards in pre-market trading.