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AAV5-RPGR · 1 trial · 1 indication
The primary outcome is defined as any of the below occurring during the 9 weeks following administration, at least possibly related to the Advanced Therapy Investigational Medicinal Product (ATIMP), not surgery alone: * Reduction in visual acuity by 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters or more * Severe unresponsive inflammation * Infective endophthalmitis * Ocular malignancy * Grade III or above non-ocular Suspected Unexpected Serious Adverse Reaction (SUSAR)
| Arm | Type | Description |
|---|---|---|
| Phase 1 (Part 1, Dose Escalation) | EXPERIMENTAL | Participants receive one of three doses of AAV5-RPGR |
| Phase 2 (Part 2; Expansion) | EXPERIMENTAL | Participants receive one of two doses of AAV5-RPGR |
| Name | Type | Description |
|---|---|---|
| AAV5-RPGR | GENETIC | Single, subretinal administration of AAV5-RPGR |
Key inclusion Criteria: * Males aged 5 years or older * Have X-linked retinitis pigmentosa confirmed by a retinal specialist (CI or PI) Key exclusion Criteria: • Have participated in another research study involving an investigational medicinal therapy for ocular disease within the last 6 months
AAV5-RPGR is an investigational gene therapy being developed for X-Linked Retinitis Pigmentosa, a rare inherited retinal disease that causes progressive vision loss. It is designed to deliver a functional version of the RPGR gene to retinal cells. The therapy is currently in Phase 1 clinical development and is not yet approved by regulatory authorities.
AAV5-RPGR targets the RPGR gene, which is mutated in X-Linked Retinitis Pigmentosa. The therapy uses an adeno-associated virus serotype 5 (AAV5) vector to deliver a functional copy of the RPGR gene to retinal cells, aiming to restore or preserve vision. This gene replacement approach is designed to address the underlying genetic cause of the disease.
AAV5-RPGR is being developed by MeiraGTx Holdings plc, a biopharmaceutical company listed on the NASDAQ under the ticker symbol MGTX. The company focuses on gene therapies for rare diseases, including ocular conditions. MeiraGTx is conducting clinical trials to evaluate the safety and efficacy of AAV5-RPGR in patients with X-Linked Retinitis Pigmentosa.
AAV5-RPGR is in Phase 1 clinical development. A Phase 1 trial has been completed, with 49 male participants enrolled. The trial was randomized and controlled, but not double-blinded. The therapy remains investigational and has not received regulatory approval. Further clinical studies would be needed to establish its safety and effectiveness.
AAV5-RPGR has been studied in one completed Phase 1 clinical trial registered as NCT03252847. The trial, titled 'Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR)', enrolled 49 male participants aged 5 years and older. It was conducted in the United States and United Kingdom and evaluated the therapy in patients with X-Linked Retinitis Pigmentosa.
AAV5-RPGR is a specific gene therapy product that delivers the RPGR gene using an AAV5 vector. It is designed for X-Linked Retinitis Pigmentosa, a condition caused by mutations in the RPGR gene. While other RPGR gene therapies may exist, AAV5-RPGR refers specifically to MeiraGTx's investigational product in clinical development.