What is AAV OPTIRPE65 used for?

AAV OPTIRPE65 is indicated for Leber congenital amaurosis, Retinitis pigmentosa.

Who makes AAV OPTIRPE65?

AAV OPTIRPE65 is developed by MeiraGTx UK II Ltd.

Last reviewed 2026-04-23 · How we verify

AAV OPTIRPE65

MeiraGTx UK II Ltd · Phase 2 active Biologic

Gene therapy targeting RPE65

Gene therapy targeting RPE65 Used for Leber congenital amaurosis, Retinitis pigmentosa.

At a glance

Generic name	AAV OPTIRPE65
Sponsor	MeiraGTx UK II Ltd
Drug class	Gene therapy
Target	RPE65
Modality	Biologic
Therapeutic area	Ophthalmology
Phase	Phase 2

Mechanism of action

AAV OPTIRPE65 is a gene therapy that uses adeno-associated virus (AAV) to deliver a functional copy of the RPE65 gene to the retina, treating inherited retinal dystrophies.

Approved indications

Leber congenital amaurosis
Retinitis pigmentosa

Common side effects

No common side effects on file.

Key clinical trials

Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65)

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

Source	Used for
ClinicalTrials.gov	Trial enrolment, design, endpoints, results

Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

AAV OPTIRPE65 CI brief — competitive landscape report
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