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SRP-9003

Phase 3

Limb-girdle Muscular Dystrophy | Monoclonal antibody | Rare Disease |Sarepta Therapeutics, Inc.|Last Updated: Mar 2, 2026

Target and mechanism

Molecular targetsarcoglycan
Target classProtein
ModalityMonoclonal antibody

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMC
Total Trials1
Total Enrollment17

FDA Designations

No designations recorded

Clinical trial landscape

SRP-9003 · 2 trials · 2 indications

Phase 3 1Phase 1 1
NCT06246513A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4Limb-girdle Muscular Dystrophy
ACTIVE NOT_RECRUITING17 Analytics
PHASE3ACTIVE NOT_RECRUITING
A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4
Limb-girdle Muscular Dystrophy
NCT0624651317
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Study Endpoints

Primary Endpoints

Cohort 1: Change from Baseline in β-SG Expression at Day 60 Post-dose as Measured by Immunofluorescence (IF) Percent β-SG Positive Fibers
Baseline, Day 60
Number of Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs)
Baseline up to Month 60
Change from Baseline in β-SG Protein Expression Quantity Assessed by Immunofluorescence (IF) Fiber Intensity at Day 60
Baseline, Day 60
Change from Baseline in β-SG Protein Expression Quantity Assessed by IF Percent Protein Fibers (PPF) at Day 60
Baseline, Day 60
Change from Baseline in β-SG Protein Expression Quantity Assessed by Western Blot at Day 60
Baseline, Day 60

Secondary Endpoints

Cohort 1 and Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by Western Assay
Baseline, Day 60
Cohort 1 and Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by IF Percent Fluorescent Intensity (PFI)
Baseline, Day 60
Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by IF Percent β-SG Positive Fibers (PβSGPF)
Baseline, Day 60
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
SRP-9003EXPERIMENTALParticipants will receive a single intravenous (IV) infusion of SRP-9003.

Interventions

NameTypeDescription
SRP-9003BIOLOGICALSolution for single IV infusion
GlucocorticoidDRUGOral tablet (prophylactic)
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Eligibility Criteria

Age Range4 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites10

Inclusion Criteria: * Cohort 1, only ambulatory participants: * Able to walk without assistive aid * 10MWR \<30 seconds * NSAD ≥25 * Cohort 2, only non-ambulatory participants: * 10MWR ≥30 seconds or unable to perform * PUL 2.0 entry scale score ≥3 * Participants must possess 1 homozygo...

Countries:United StatesBelgiumGermanyItalySpainUnited Kingdom
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Frequently asked questions about SRP-9003

What is SRP-9003 used for?

SRP-9003, also known as bidridistrogene xeboparvovec, is an investigational gene therapy being developed for the treatment of Limb Girdle Muscular Dystrophy, specifically Type 2E/R4, which is caused by beta-sarcoglycan deficiency. It is designed to address the underlying genetic cause of this rare muscle-wasting disease.

What does SRP-9003 target?

SRP-9003 targets the sarcoglycan protein, specifically the beta-sarcoglycan subunit. The therapy is designed to deliver a functional copy of the beta-sarcoglycan gene to muscle cells, aiming to restore the production of this protein, which is deficient in patients with Limb Girdle Muscular Dystrophy Type 2E/R4.

Who is developing SRP-9003?

SRP-9003 is being developed by Sarepta Therapeutics, Inc., a biopharmaceutical company focused on rare genetic diseases. Sarepta's stock is traded under the ticker symbol SRPT on the NASDAQ exchange.

What phase is SRP-9003 in?

SRP-9003 is currently in clinical development. It has completed Phase 1 trials and is now being evaluated in a Phase 3 clinical trial. The drug is investigational and has not yet been approved by regulatory authorities such as the FDA.

What clinical trials is SRP-9003 in?

SRP-9003 is being studied in two active clinical trials. NCT05876780 is a Phase 1 study evaluating safety, tolerability, and efficacy in participants with Limb Girdle Muscular Dystrophy Type 2E/R4. NCT06246513 is a Phase 3 trial testing the therapy as a possible treatment for the same condition, with sites in the US and Europe.

Is SRP-9003 the same as bidridistrogene xeboparvovec?

Yes, SRP-9003 is also known as bidridistrogene xeboparvovec. Both names refer to the same investigational gene therapy being developed by Sarepta Therapeutics for Limb Girdle Muscular Dystrophy Type 2E/R4.