This project has been flagged by a community member as inappropriate due to reason below.*

Expert Interview

Slingshot members are talking to an expert! The topic is:

Examining Phase 1/2 PM359 Results in Chronic Granulomatous Disease from Prime Medicine

Ticker(s): PRME

Who's the expert?

A 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.

Interview Questions
Q1.

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_insights
Q2.

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?

Added By: slingshot_insights
Q3.

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?

Added By: slingshot_insights
Q4.

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?

Added By: slingshot_insights
Q5.

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?

Added By: slingshot_insights
Q6.

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?

Added By: slingshot_insights
Q7.

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?

Added By: slingshot_insights
Q8.

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?

Added By: slingshot_insights

Are You Interested In These Questions?

Slingshot Insights Explained
209Days Left to Join Project

Expert research benefits investors by giving them timely access to unbiased real world perspectives on highly specialized topics. Slingshot Insights' crowdfunded model makes this access available at a fraction of the cost of other expert networks.

Reason

*Slingshot Insights provides access to information, not investment advice. We work to support you and facilitate access to experts; however we are not responsible for monitoring calls for the disclosure of MNPI. You should obtain financial, legal and tax advice from your qualified and licensed advisers before deciding to invest in any security.