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BEAM-103

Phase 1

Healthy Subjects | Small molecule | Other |Beam Therapeutics Inc.|Last Updated: Dec 26, 2025

Target and mechanism

Molecular targetCD117
Target classProtein
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

RandomizedPLACEBO_CONTROLLED
Total Trials1
Total Enrollment30

FDA Designations

No designations recorded

Clinical trial landscape

BEAM-103 · 1 trial · 2 indications

Phase 1 1
NCT07304791This is a Phase 1, Randomized, Single-blind, Placebo-controlled Study to Assess the Safety, Pharmacokinetics (PK), and Pharmacodynamics (PD) of BEAM-103 in Healthy SubjectsHealthy Subjects
RECRUITING30 Analytics
PHASE1RECRUITING
This is a Phase 1, Randomized, Single-blind, Placebo-controlled Study to Assess the Safety, Pharmacokinetics (PK), and Pharmacodynamics (PD) of BEAM-103 in Healthy Subjects
Healthy SubjectsUnlock trial analytics

Study Endpoints

Primary Endpoints

To determine the safety and tolerability of single ascending doses of BEAM-103
from enrollment to the end of study at 5 months

Incidence of treatment-emergent AEs, serious AEs, severe AEs, AEs related to BEAM-103

Secondary Endpoints

Change in Free Stem Cell Factor (SCF) in Peripheral Blood Over Time
from the start of treatment to the end of study at 5 months
Change in Tryptase Level in Peripheral Blood Over Time
from the start of treatment to the end of study at 5 months
Change in Reticulocyte Count in Peripheral Blood Over Time
From enrollment to end of study at 5 months
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Study Design & Arms

AllocationRANDOMIZED
MaskingSINGLE
ModelSEQUENTIAL
PurposeBASIC_SCIENCE

Treatment Arms

ArmTypeDescription
BEAM-103 Drug ProductEXPERIMENTALBEAM-103 is a monoclonal antibody that will be administered in single dose ascending dose cohorts as an intravenous infusion through the arm.
PlaceboPLACEBO_COMPARATORNormal Saline Solution will be administered as a matching place in single dose ascending dose cohorts as an intravenous infusion through the arm.

Interventions

NameTypeDescription
BEAM-103DRUG• BEAM-103 is a monoclonal antibody administered as a single intravenous dose
Placebo ComparatorDRUG• The matching placebo comparator is administered as a single intravenous dose
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Eligibility Criteria

Age Range18 Years to 55 Years
SexALL
Healthy VolunteersYes
Study Sites1

Inclusion Criteria: * Ages 18 to ≤55 years * Body mass index (BMI) of 18.5 to 25 kg/m2 * Morning neutrophil count ≥2.6 × 109 cells/L for Black/African American subjects or ≥3.5 ×109 cells/L for Caucasian/Other subjects * Baseline hemoglobin value of ≥14 g/dL for males and ≥12 g/dL for females * Bas...

Countries:United Kingdom
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Frequently asked questions about BEAM-103

What is BEAM-103 used for?

BEAM-103 is an investigational small molecule being studied for use in healthy subjects. It is currently in a Phase 1 clinical trial to assess its safety, pharmacokinetics, and pharmacodynamics in healthy participants. The drug is not yet approved and remains in clinical development.

What does BEAM-103 target?

BEAM-103 targets CD117, a protein also known as c-Kit. This target is a receptor tyrosine kinase involved in cell signaling. The drug is designed to interact with this protein, though its full mechanism of action is still under investigation in clinical trials.

Who makes BEAM-103?

BEAM-103 is being developed by Beam Therapeutics Inc., a biopharmaceutical company traded on the stock exchange under the ticker symbol BEAM. The company is conducting a Phase 1 clinical trial of this investigational drug in healthy subjects.

What phase is BEAM-103 in?

BEAM-103 is in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by regulatory authorities. The ongoing Phase 1 trial is recruiting participants to evaluate the drug's safety, pharmacokinetics, and pharmacodynamics in healthy subjects.

What clinical trials is BEAM-103 in?

BEAM-103 is being studied in one active Phase 1 clinical trial with the identifier NCT07304791. This is a randomized, single-blind, placebo-controlled study enrolling 30 healthy subjects in the United Kingdom. The trial is currently recruiting participants aged 18 years and older.