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Low dose LX2006

Phase 1

Friedreich Ataxia | Gene therapy | Neurology |Lexeo Therapeutics, Inc.|Last Updated: Dec 23, 2025

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDDMC
Total Trials1
Total Enrollment8
FDA Designations
BREAKTHROUGH_THERAPYRMATORPHAN_DRUGFAST_TRACKRARE_PEDIATRIC_DISEASE
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05445323Gene Therapy for Cardiomyopathy Associated With Friedreich's AtaxiaPHASE1 ACTIVE NOT_RECRUITING 8 — — Aug 24, 2022Sep 1, 2029Dec 23, 20253 United States
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Study Endpoints
Primary Endpoints
Treatment-emergent adverse events (TEAEs) and Treatment-emergent serious events (TESAEs)
Change from baseline to end of year 5 post dose
Secondary Endpoints
Change from baseline in LVMi
Change from baseline to end of year 5 post dose
Change from baseline in LVEF
Change from baseline to end of year 5 post dose
Change from baseline in cardiac fibrosis as measured by cardiac MRI
Change from baseline to end of year 5 post dose
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Cohort 1/ Cohort 2/ Cohort 3EXPERIMENTAL -
Interventions
NameTypeDescription
Low dose LX2006GENETICAdeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN)
Mid Dose LX2006GENETICAdeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN)
High Dose LX2006GENETICAdeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN)
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Eligibility Criteria
Age Range18 Years — 50 Years
SexALL
Healthy VolunteersNo
Study Sites3

Inclusion Criteria: * Confirmed genetic diagnosis of FA, with onset being before 25 years of age * Protocol specified ranges for antibodies * Protocol specified measures of FA cardiomyopathy Exclusion Criteria: * Protocol specified ranges for left ventricular ejection fraction (LVEF) as measured ...

Countries:United States
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT05445323primaryCompletionDate: changed
LOWMay 24, 2026NCT05445323studyFirstPostDate: changed