The DHR assay showed 58% oxidase activity by Day 15 and 66% by Day 30—far exceeding the 20% clinical threshold. What does this speed and depth of response suggest about the efficiency of Prime Editing compared to current gene therapies for CGD?

Neutrophil engraftment occurred by Day 14 and platelet engraftment by Day 19—almost twice as fast as median values seen in traditional gene therapies. How clinically significant is this rapid engraftment for reducing infection risk and hospital stay duration?

PM359 was well-tolerated with no serious AEs attributed to the product. In the context of gene-edited autologous therapies, how reassuring is this safety profile, and what additional safety signals would you be monitoring as the trial expands?

PM359 is the first clinical application of Prime Editing in humans. How do you evaluate the specificity and precision of Prime Editing compared to CRISPR-Cas9 or base editing in the context of hematopoietic stem cell modification?

Given that PM359 corrects the delGT mutation in NCF1, how closely do functional outcomes like DHR positivity correlate with long-term clinical stability in CGD, and what longitudinal data would you need to consider the therapy curative?

While PM359 targets the p47phox variant, how feasible is it to adapt Prime Editing strategies for other CGD genotypes, such as X-linked forms, and what challenges would you anticipate in extending this approach?

This is a one-time curative approach. How might this shift the paradigm for treatment of rare inherited disorders—especially in pediatric populations—both clinically and ethically, particularly regarding early intervention?

Given Prime Medicine’s decision to seek external partners for PM359’s continued development, what are the critical factors that will determine whether this therapy reaches market, and how might this impact investor and patient confidence in Prime Editing broadly?