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LX101

Phase 1

Inherited Retinal Dystrophy Associated With RPE65 Mutations | Gene therapy | Rare Disease |Co-Diagnostics, Inc.|Last Updated: Mar 6, 2026

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
CONTROLLEDBiomarker
Total Trials1
Total Enrollment9
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06196827Safety and Tolerability of LX101 for Inherited Retinal Dystrophy Associated With RPE65 MutationsPHASE1 ACTIVE NOT_RECRUITING 9 — — Jul 2, 2022Dec 1, 2027Mar 6, 20262 China
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Study Endpoints
Primary Endpoints
Incidence of adverse events (AEs) and serious adverse events (SAEs)
12 months

Incidence of ocular and non-ocular AEs and SAEs following LX101 subretinal injection

Incidence of dose-limiting toxicity (DLT)
1 month

Incidence of DLT following LX101 subretinal injection at different doses

Secondary Endpoints
Efficacy of LX101 in study eye
12 months
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
LX101 Dose 1EXPERIMENTAL -
LX101 Dose 2EXPERIMENTAL -
Interventions
NameTypeDescription
LX101GENETICSubretinal Administration
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Eligibility Criteria
Age Range6 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites2

Inclusion Criteria: Subject and/or their guardian signing a written informed consent. Diagnosed with biallelic RPE65 mutation-associated inherited retinal dystrophy. Subjects are 6 years of age or older. Visual acuity of ≤ 20/63 or visual field less than 20 degrees in the eye to be injected. Ex...

Countries:China
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