Ed­i­tas' CRISPR-based eye ther­a­py proves safe, but the ju­ry's still out on whether it works

Re­searchers at Ed­i­tas Med­i­cine re­vealed long-an­tic­i­pat­ed da­ta for their CRISPR gene edit­ing pro­gram Wednes­day morn­ing, the sec­ond-ever batch of hu­man re­sults for in vi­vo ad­min­is­tra­tion of the No­bel Prize-win­ning tech­nol­o­gy. And while the ther­a­py ap­peared re­mark­ably safe, a huge step for­ward in its own right, ex­perts ques­tioned whether the ear­ly ef­fi­ca­cy fig­ures will ul­ti­mate­ly bear fruit.

Ed­i­tas’ da­ta come from a Phase I/II study eval­u­at­ing a gene edit­ing can­di­date for LCA10, a form of in­her­it­ed blind­ness, with the first cut look­ing at six adult pa­tients across two low- and mid-dose co­horts. There were no dose-lim­it­ing tox­i­c­i­ties and most ad­verse events stemmed from the sur­gi­cal pro­ce­dure used to in­ject the treat­ment in­to the eye, Ed­i­tas said. It was safe enough for the in­de­pen­dent da­ta mon­i­tor­ing com­mit­tee to rub­ber stamp test­ing the ex­per­i­men­tal drug in chil­dren.

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.

SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER