Graphite Bio Gets Fast Track Designation for GPH101 Sickle Cell Treatment

By Chris Wack

Graphite Bio Inc. said Tuesday that the U.S. Food and Drug Administration has granted fast track designation to GPH101 for the treatment of sickle cell disease.

The company said that GPH101 is an investigational next-generation gene-edited autologous hematopoietic stem cell therapy designed to directly correct the genetic mutation that causes SCD.

Graphite Bio said it is continuing to enroll patients in its Phase 1/2 trial, and expects to dose its first patient later this year, with initial proof-of-concept data anticipated next year.

The FDA's fast track program speeds up the development and review of new drugs or biologics that are intended to treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs. GPH101 was previously granted orphan drug designation by the FDA.

Graphite Bio shares were up 9% to $4.36 in premarket trading.

Write to Chris Wack at [e-mail]

(END) Dow Jones Newswires

05-03-22 0835ET