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NCT06559176 (PM359 in Chronic Granulomatous Disease): BVCT ex-ante prediction vs. reported outcome

On 13 May 2025, BioinvestGPT's BVCT predicted technical success and commercial partial success for this trial. The 19 May 2025 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: statistically probablysignificant and commercially probably insufficient.

Clinical Trial

NCTID
NCT06559176
Drug Name
PM359
Drug MoA
prime-editor-mediated MCF1-delGT-mutation-corrected CD34+ autologous hematopoietic stem cells
Drug Modality
cell therapy
Drug Class
First-In-Class
Therapeutic Area
Immune System Diseases
Indication
Chronic Granulomatous Disease (CGD)
Human Patients
12
Sponsor
Prime Medicine
Phase
Phase 1 Phase 2

Prediction of , index 2021

Prospective Prediction
Prediction Index
2021
Prediction Date
Commercial Prediction
partial SUCCESS (commercially maybe sufficient yet moderate clinical benefit in OS/infection frequency reduction/active infection resolution/GvHD/autoimmune process frequency/active autoimmune process resolution non-superior to interferon gamma)
Technical Prediction
SUCCESS (statistically significant clinical benefit in OS/infection frequency reduction/active infection resolution/GvHD/autoimmune process frequency/active autoimmune process resolution compared with placebo)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
6 days in advance
Readout Data Interpretation
PM359’s further development has been discontinued probably due to insufficient benefit-risk profile
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically probablysignificant and commercially probably insufficient
Prediction Classification
True Negative (TN)

ClinicalTrials.gov record

ClinicalTrials.gov record, retrieved 2026-09-24. Third-party data.

Official title
A Phase 1/2 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Modified Ex Vivo Using Prime Editing (PM359) in Participants With Autosomal Recessive Chronic Granulomatous Disease Due to Mutations in the NCF1 Gene
Phase
Phase 1, Phase 2
Status
Enrolling by invitation
Enrollment
12
Lead sponsor
Prime Medicine, Inc.
Conditions
Chronic Granulomatous Disease; Granulomatous Disease, Chronic
Interventions
BIOLOGICAL: PM359
Design
Single group · None
Primary purpose
Treatment
Start
2024-10-17
Primary completion
2030-01
Completion
2030-02
Primary outcomes
  • 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)

NCT06559176 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

How to read these records: evidence · methodology · all trials