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PM647a

Phase 1

Alpha-1 Antitrypsin Deficiency | Small molecule | Rare Disease |Prime Medicine, Inc.|Trials Updated: Oct 21, 2026

PM647a development status

Highest phase Phase 1
Registered trials 1 across 1 sponsor since Oct 2026

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMC
Total Trials1
Total Enrollment54

FDA Designations

No designations recorded

PM647a clinical trials

PM647a · 1 trial · 4 indications

Phase 1 1
NCT07887347A Study of the Safety and Efficacy of Prime Editing (PM647a) in Participants With Alpha-1 Antitrypsin DeficiencyAlpha-1 Antitrypsin Deficiency
NOT YET_RECRUITING54 Analytics
PHASE1NOT YET_RECRUITING
A Study of the Safety and Efficacy of Prime Editing (PM647a) in Participants With Alpha-1 Antitrypsin Deficiency
Alpha-1 Antitrypsin Deficiency
NCT0788734754
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Study Endpoints

Primary Endpoints

Safety and tolerability of PM647a. Quantified by frequency and severity of treatment emergent adverse events (TEAEs)
Immediately post-infusion up to Week 48

Secondary Endpoints

Frequency and severity of Dose Limiting Toxicities (DLTs)
Infusion up to and including 28 days
Percentage of participants with ≥ 11 µM total serum alpha-1 antitrypsin (AAT) levels
Week 12 post-infusion
Percentage of participants with ≥ 11 µM total serum AAT levels
Week 4, Week 6, Week 12, Week 24 and Week 48 post-infusion
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
PM647aEXPERIMENTALPM647a is a sterile suspension of lipid nanoparticle (LNP)-formulated Prime Editors (PEs) intended for single dose intravenous (IV) infusion for the treatment of Alpha-1 Antitrypsin Deficiency (AATD)

Interventions

NameTypeDescription
PM647aDRUGPM647a is being evaluated in participants with Alpha-1 Antitrypsin Deficiency (AATD) caused by a homozygous Pi\*Z allele (Pi\*ZZ genotype), with an observed lung phenotype (Group A), or an observed liver and lung or liver alone phenotype (Group B)
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo

Inclusion Criteria: * Participants at least 18 years of age at the time of consent and able to provide written consent * Confirmed diagnosis of AATD as determined by medical history consistent with AATD, and diagnostic genetic testing performed by the designated central laboratory demonstrating hom...

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Recent Changes (Last 90 Days)

LOWOct 21, 2026NCT07887347NEW_TRIAL: changed
LOWOct 21, 2026NCT07887347NEW_TRIAL: changed
LOWOct 21, 2026NCT07887347NEW_TRIAL: changed

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