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CLINICAL UPDATE | Graphite’s Hopes For Sickle Cell Cure Blunted After First Patient Dosed Experiences Serious Event

Graphite Bio has hit an unexpected roadbock to the clinical development of its experimental gene-editing therapy for sickle cell disease (SCD) nula-cel, as an adverse event caused the biotech to pause its lead programme.

The company cited a “serious adverse event in the first patient dosed” with its gene therapy nulabeglogene autogedtemcel (nula-cel) as part of a phase 1/2 trial for SCD. The event is likely to be related to the treatment, the company concluded in a press release.

Specifically, a patient experienced prolonged low blood cell counts, requiring ongoing transfusion and growth factor support. Graphite reported the incident to the FDA and in the meantime has voluntarily paused the trial.

While Graphite said the event did not meet the requirements to halt the study, both the clinical investigators and the trial’s safety monitoring committee agreed with the decision to suspend the dosing of additional patients while the company tries to find out what happened.

“We are committed to working closely with our scientific and clinical experts to fully assess this event and identify a potential path to resume the CEDAR study,” CEO Josh Lehrer, M.D., said in a postmarket release last Thursday. “We are grateful for the partnership with the sickle cell community, our clinical investigators, our founders and scientific experts and the FDA as we determine next steps for our nula-cel programme in sickle cell disease.”

The adverse event derails both Graphite’s clinical and financial strategies. Plans for β-thalassaemia treatment GPH102 to enter the clinic by mid-2024 no longer seem likely, admitted the company, which is now working to “identify operational efficiencies to extend its cash position to at least 2026.”

Read Graphite Bio’s Full Press Release HERE

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