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BEAM-302

Phase 1

Alpha 1-Antitrypsin Deficiency | Small molecule | Rare Disease |Beam Therapeutics Inc.|Last Updated: Mar 20, 2026

Target and mechanism

Molecular targetSERPINA1 PiZ
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMC
Total Trials1
Total Enrollment106

FDA Designations

ORPHAN_DRUGRMAT

Clinical trial landscape

BEAM-302 · 1 trial · 1 indication

Phase 1 1
NCT06389877A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)Alpha 1-Antitrypsin Deficiency
RECRUITING106 Analytics
PHASE1RECRUITING
A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)
Alpha 1-Antitrypsin Deficiency
NCT06389877106
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Study Endpoints

Primary Endpoints

Phase 1 Dose Exploration: Rates of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
2 years

Numbers and percentages of patients reporting a given AE

Phase 2 Dose Expansion: Absolute blood levels of total AAT
2 Years

Absolute Levels of AAT over time

Secondary Endpoints

Phase 1 Dose Exploration: Absolute blood levels of total AAT
2 Years
Phase 2 Dose Expansion: Rates of TEAEs and SAEs
2 Years
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
BEAM-302 Drug ProductEXPERIMENTAL -

Interventions

NameTypeDescription
BEAM-302DRUGBEAM-302 is a lipid nanoparticle (LNP)-based therapy for the treatment of patients with AATD
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Eligibility Criteria

Age Range18 Years to 70 Years
SexALL
Healthy VolunteersNo
Study Sites11

Part A: Inclusion Criteria: * Males or females 18 - 70 years of age inclusive at the time of consent. * Diagnosis of AATD and homozygous for the PiZZ mutation (confirmed by genetic testing). * Blood total AAT level \<11 μM or equivalent protein in mg/dL. * Patients receiving augmentation therapy i...

Countries:United StatesAustraliaIrelandNetherlandsNew ZealandUnited Kingdom
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Frequently asked questions about BEAM-302

What is BEAM-302 used for?

BEAM-302 is an investigational small molecule being developed for the treatment of Alpha 1-Antitrypsin Deficiency (AATD), a rare genetic condition. It is currently in Phase 1 clinical development and has not been approved by the FDA.

What does BEAM-302 target?

BEAM-302 targets the SERPINA1 PiZ gene, which is the mutated form of the SERPINA1 gene responsible for Alpha 1-Antitrypsin Deficiency. By targeting this specific genetic mutation, the therapy aims to address the underlying cause of the disease.

Who is developing BEAM-302?

BEAM-302 is being developed by Beam Therapeutics Inc., a biotechnology company publicly traded under the ticker symbol BEAM. The company is conducting clinical trials to evaluate the safety and efficacy of this investigational therapy.

What phase is BEAM-302 in?

BEAM-302 is currently in Phase 1 clinical development. It is an investigational drug, meaning it has not yet received FDA approval and is still being studied in clinical trials to assess its safety and effectiveness in patients.

What clinical trials is BEAM-302 in?

BEAM-302 is being evaluated in a Phase 1 clinical trial with the identifier NCT06389877. This study is recruiting adult patients with Alpha 1-Antitrypsin Deficiency and is being conducted in the United States, Australia, Ireland, Netherlands, New Zealand, and the United Kingdom.

Has BEAM-302 received any FDA designations?

Yes, BEAM-302 has received Orphan Drug designation and Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA. These designations are intended to support the development of therapies for serious or life-threatening conditions.