Eli Lilly removes Gaucher disease gene therapy trial from pipeline, citing unmet internal criteria

Eli Lilly has announced the removal of a phase 1/2 study testing its GBA1 gene therapy for Gaucher disease type 1. The program was part of the assets acquired when the company bought Prevail Therapeutics for $1 billion.

The decision follows an earlier cut of a different gene therapy aimed at frontotemporal dementia, leaving the GBA1 therapy for Parkinson's disease as the sole remaining candidate from the acquisition.

The halted trial was set to enroll roughly 15 adult patients to evaluate safety and tolerability across up to three dose levels. No safety signals were reported, but the results did not meet Lilly’s internal success criteria.

Lilly said it will keep advancing the GBA1 gene therapy in Parkinson's disease through the ongoing Propel trial, indicating continued belief in the target despite the setback in Gaucher disease.