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PM359

Phase 1

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

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMC
Total Trials1
Total Enrollment12

FDA Designations

RMATFAST_TRACKORPHAN_DRUGRARE_PEDIATRIC_DISEASE

Clinical trial landscape

PM359 · 1 trial · 2 indications

Phase 1 1
NCT06559176A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD )Chronic Granulomatous Disease
ENROLLING BY_INVITATION12 Analytics
PHASE1ENROLLING BY_INVITATION
A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD )
Chronic Granulomatous Disease
NCT0655917612
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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 to 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

Frequently asked questions about PM359

What is PM359 used for?

PM359 is an investigational therapy being developed for Chronic Granulomatous Disease (CGD), specifically for patients with p47phox autosomal recessive CGD. It is currently in Phase 1 clinical development and has not been approved by the FDA.

What does PM359 target?

PM359 is a gene editing therapy that uses prime editing technology to correct the genetic mutation responsible for p47phox autosomal recessive Chronic Granulomatous Disease. It is designed to restore the function of the p47phox protein in immune cells.

Who makes PM359?

PM359 is being developed by Prime Medicine, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol PRME. The company is conducting a Phase 1 clinical trial to evaluate the safety and efficacy of PM359 in patients with Chronic Granulomatous Disease.

What phase is PM359 in?

PM359 is currently in Phase 1 clinical development. It is an investigational therapy and has not received FDA approval. The ongoing Phase 1 trial is enrolling patients by invitation and is being conducted in the United States, Canada, and the United Kingdom.

What clinical trials is PM359 in?

PM359 is being studied in a Phase 1 clinical trial registered as NCT06559176. This trial is evaluating the safety and efficacy of PM359 in participants with p47phox autosomal recessive Chronic Granulomatous Disease. The study is enrolling 12 participants aged 6 years and older.

What FDA designations has PM359 received?

PM359 has received several FDA designations, including Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations. These designations are intended to expedite the development and review of therapies for serious conditions.