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4D-125

Gene therapy (AAV vector) · Gene replacement

Phase 1/2 Most advanced trial
1 Registered trials

What is 4D-125?

4D-125 is a gene therapy built from an AAV capsid variant (4D-R100) carrying a codon-optimized human RPGR transgene. It is studied in X-linked retinitis pigmentosa, an inherited eye disease.

Summary written with AI from the sources listed at the end of this page.

At a glance

Drug type
Gene therapy (AAV vector)
Mechanism
Gene replacement
Therapy area
Ophthalmology
In trials of
FDMT
FDA status
Investigational, not approved in the US. Most advanced trial: Phase 1/2

🧪 Clinical trials

TrialPhaseConditionStatusSponsor
NCT04517149 Phase 1/2 X-Linked Retinitis Pigmentosa Active not recruiting FDMT

Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.

Conditions studied

X-Linked Retinitis Pigmentosa

Sources

Updated Oct 11, 2026.

Data: FDA (Drugs@FDA, Purple Book, DailyMed), NIH (NCI Thesaurus, GSRS, RxNorm), ClinicalTrials.gov • Informational purposes only, not medical or investment advice. • All drugs A–Z

This data is for informational purposes only, not investment advice. BioRadar does not provide buy/sell recommendations. Past performance does not guarantee future results. Always do your own due diligence.