Reni-cel Clinical Trials Progress
Completed adolescent cohort enrollment for the RUBY trial, and adult cohort enrollment in the EdiTHAL trial. Clinical data from RUBY and EdiTHAL trials show promising results with all patients free from vaso-occlusive events and a high level of fetal hemoglobin.
In Vivo Pipeline Advancements
On track to establish in vivo preclinical proof of concept for an undisclosed indication by the end of the year. Strengthening in vivo discovery capabilities and pursuing a differentiated strategy for gene editing.
Financial Position
Cash, cash equivalents, and marketable securities as of June 30 were $318 million, expected to fund operations into 2026.
Manufacturing Improvements
Robust manufacturing process for reni-cel with a low failure rate, potentially reducing patient burden and cost.