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SRP-9003

Phase 3

Limb Girdle Muscular Dystrophy | Gene therapy | Rare Disease |Sarepta Therapeutics, Inc.|Trials Updated: Mar 2, 2026

SRP-9003 development status

Highest phase Phase 3
Registered trials 3 across 1 sponsor since Aug 2018

SRP-9003 target and mechanism

Molecular targetsarcoglycan
Target classProtein
ModalityGene therapy

Also known as scAAVrh74.MHCK7.hSGCB

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMC
Total Trials2
Total Enrollment23

FDA Designations

No designations recorded

SRP-9003 clinical trials

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 is an investigational gene therapy being developed for limb girdle muscular dystrophy, specifically the type 2E/R4 subtype caused by beta-sarcoglycan deficiency. It is not yet approved for any indication and remains in clinical development.

What does SRP-9003 target?

SRP-9003 targets sarcoglycan, a protein complex critical for muscle membrane stability. In limb girdle muscular dystrophy type 2E/R4, mutations in the beta-sarcoglycan gene lead to loss of this protein. SRP-9003 aims to deliver a functional copy of the gene to restore sarcoglycan expression in muscle cells.

Who is developing SRP-9003?

SRP-9003 is being developed by Sarepta Therapeutics, Inc., which trades on the Nasdaq under the ticker SRPT. Sarepta is a biopharmaceutical company focused on rare neuromuscular diseases.

What phase is SRP-9003 in?

SRP-9003 is in Phase 3 clinical development. A Phase 3 trial is currently active but not recruiting participants. The program also includes a Phase 1 study. SRP-9003 is investigational and has not been approved by the FDA.

What clinical trials is SRP-9003 in?

SRP-9003 is being evaluated in two active clinical trials: a Phase 3 study (NCT06246513) in participants with limb girdle muscular dystrophy type 2E/R4, and a Phase 1 study (NCT05876780) in ambulatory and non-ambulatory participants with the same condition. Both trials are active but not recruiting.

Is SRP-9003 the same as bidridistrogene xeboparvovec?

Yes, SRP-9003 is also known as bidridistrogene xeboparvovec. The two names refer to the same investigational gene therapy developed by Sarepta Therapeutics for limb girdle muscular dystrophy type 2E/R4.

Other drugs targeting sarcoglycan

SRP-9004 SRPT· PHASE1

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