Examining Clinical Trial Progress & Adaptive Strategies for Safer In Vivo Gene Editing

• Sharing early clinical data from ongoing patients dosing, accelerating confidence in therapeutic potential
• Implementing adaptive cohort designs and monitoring frameworks, improving trial flexibility and patient safety
• Aligning eligibility and dosing strategies with evolving biomarker insights, ensuring efficient global progression 

Speakers:

George Diaz

Vice President - Urea Cycle Disorders & Therapeutic Area Lead

iECURE