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PM359

Phase 1

Chronic Granulomatous Disease | Monoclonal antibody | Other |Prime Medicine, Inc.|Last Updated: Apr 6, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment12
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06559176A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD )PHASE1 ENROLLING BY_INVITATION 12 — — Oct 17, 2024Feb 1, 2030Apr 6, 20265 United States, Canada +1
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Study Endpoints
Primary Endpoints
Safety of administration of PM359, as quantified by frequency of adverse events (AEs) after drug product infusion
PM359 infusion through Month 12 after PM359 infusion
Percentage of participants with sustained reconstitution of NADPH oxidase activity in neutrophils
At Month 6 and Month 12 after PM359 infusion, as compared to baseline
Secondary Endpoints
Frequency of all drug product-related AEs, ≥ Grade 3 AEs, and serious adverse events (SAEs)
Signing of ICF through Month 36 following PM359 infusion
Time to neutrophil engraftment
From PM359 infusion through engraftment, typically within 2-3 weeks but assessed up to 36 months
Transplant related mortality
From PM359 infusion, assessed at 100 Days and 1 Year post-PM359 infusion
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
PM359EXPERIMENTALPM359 is an autologous CD34+ hematopoietic stem cell (HSC) suspension that is Prime Edited at the NCF1 locus resulting in expression of the p47phox protein.
Interventions
NameTypeDescription
PM359BIOLOGICALSingle dose of PM359 administered autologously by intravenous (I.V.) infusion following myeloablative conditioning with busulfan
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Eligibility Criteria
Age Range6 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites5

Inclusion Criteria: * Autosomal recessive Chronic Granulomatous Disease due to the delGT mutation in NCF1 causing dysfunction of p47phox * Treated and followed for at least the past 2 years in a specialized center * Willingness to participate in this study as well as a long-term follow-up study wit...

Countries:United StatesCanadaUnited Kingdom
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06559176Status: ACTIVE_NOT_RECRUITING → ENROLLING_BY_INVITATION
LOWMay 24, 2026NCT06559176studyFirstPostDate: changed