Halted sickle cell gene therapy trial resumes under new sponsor
Gene-editing therapy for sickle cell disease restarts trials after a serious adverse event halted earlier study.
Adobe (Photo via STAT)
A clinical trial for a gene-editing therapy designed to treat sickle cell disease has resumed three years after it was suspended due to a serious adverse event. The study, originally known as the CEDAR trial, was paused in January 2023 by its developer, Graphite Bio, after the first patient treated with the therapy experienced prolonged pancytopenia.
The therapy, nula-cel, uses CRISPR technology to replace the faulty beta-globin gene with a copy intended to produce normal red blood cells. This approach differs from other FDA-approved gene therapies for sickle cell disease, which function by increasing anti-sickling hemoglobin rather than correcting the underlying mutation directly.
Following the 2023 pause, then-CEO of Graphite Bio, Dr. Josh Lehrer, stated that the company was committed to assessing the event and identifying a path to resume the study. At the time, the company indicated it was investigating potential manufacturing process modifications as a mitigation strategy.
The trial is now active under the name Restore and is sponsored by Kamau Therapeutics. It is currently available for adults 18 and older with severe sickle cell disease at Lucile Packard Children’s Hospital Stanford, with plans to expand eligibility to include children as young as 12.