Expert Interview
Examining Phase 1/2 PM359 Results in Chronic Granulomatous Disease from Prime Medicine
Ticker(s): PRMEA clinical immunologist or hematologist specializing in rare genetic immunodeficiencies, particularly Chronic Granulomatous Disease. Ideal candidates should have experience with gene therapy or stem cell transplantation, and be familiar with the molecular mechanisms of NADPH oxidase function and DHR assay interpretation.
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?
Added By: slingshot_insightsNeutrophil 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?
Added By: slingshot_insightsPM359 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?
Added By: slingshot_insightsPM359 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?
Added By: slingshot_insightsGiven 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?
Added By: slingshot_insightsWhile 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?
Added By: slingshot_insightsThis 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?
Added By: slingshot_insightsGiven 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?
Added By: slingshot_insightsAre You Interested In These Questions?
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