botaretigene sparoparvovec (AAV-RPGR) / MeiraGTx, J&J - LARVOL DELTA

Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene (clinicaltrials.gov) - P3 | N=97 | Completed | Sponsor: Janssen Research & Development, LLC | Active, not recruiting ➔ Completed

Gene therapy • Trial completion • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis Pigmentosa (XLRP) (clinicaltrials.gov) - P2 | N=42 | Recruiting | Sponsor: Janssen Research & Development, LLC | Not yet recruiting ➔ Recruiting

Enrollment open • Gene therapy • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked Retinitis Pigmentosa (clinicaltrials.gov) - P3 | N=4 | Recruiting | Sponsor: Janssen Pharmaceutical K.K. | Trial completion date: Feb 2030 ➔ May 2030

Trial completion date • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis Pigmentosa (XLRP) (clinicaltrials.gov) - P2 | N=42 | Not yet recruiting | Sponsor: Janssen Research & Development, LLC

Gene therapy • New P2 trial • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked Retinitis Pigmentosa (clinicaltrials.gov) - P3 | N=4 | Recruiting | Sponsor: Janssen Pharmaceutical K.K. | Trial completion date: Oct 2029 ➔ Feb 2030

Trial completion date • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Phase 1/2 AAV5-hRKp.RPGR (Botaretigene Sparoparvovec) Gene Therapy: Safety and Efficacy in RPGR-associated X-linked Retinitis Pigmentosa. (PubMed, Am J Ophthalmol) - P1/2 | "AAV5-hRKp.RPGR demonstrated an anticipated and manageable AE profile through 52 weeks. Safety and efficacy findings support investigation in a phase 3 trial."

Gene therapy • Journal • P1/2 data • Gene Therapies • Genetic Disorders • Inflammation • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Pediatrics • Retinal Disorders • Retinitis Pigmentosa • Uveitis

A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked Retinitis Pigmentosa (clinicaltrials.gov) - P3 | N=4 | Recruiting | Sponsor: Janssen Pharmaceutical K.K. | Trial completion date: May 2029 ➔ Oct 2029

Trial completion date • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene (clinicaltrials.gov) - P3 | N=97 | Active, not recruiting | Sponsor: Janssen Research & Development, LLC | Trial completion date: Feb 2025 ➔ Sep 2024

Gene therapy • Trial completion date • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

MGT-RPGR-022: Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene (clinicaltrials.gov) - P3 | N=97 | Active, not recruiting | Sponsor: Janssen Research & Development, LLC | Trial completion date: Sep 2029 ➔ Dec 2029

Gene therapy • Trial completion date • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

AAV-RPGR Gene Therapy Rescues Opsin Mislocalisation in a Human Retinal Organoid Model of RPGR-Associated X-Linked Retinitis Pigmentosa. (PubMed, Int J Mol Sci) - "In addition, treatment with AAV-RPGR restored rhodopsin localisation within RPGR-KO ROs, reducing mislocalisation to the photoreceptor outer nuclear layer. These data provide mechanistic insights into RPGRORF15 gene supplementation functional potency in human photoreceptor cells and support the previously reported Phase I/II trial positive results using this vector construct in patients with RPGR-associated XLRP, which is currently being tested in a Phase III clinical trial."

Gene therapy • Journal • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene (clinicaltrials.gov) - P3 | N=97 | Active, not recruiting | Sponsor: Janssen Research & Development, LLC | Recruiting ➔ Active, not recruiting

Enrollment closed • Gene therapy • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene (clinicaltrials.gov) - P3 | N=97 | Active, not recruiting | Sponsor: Janssen Research & Development, LLC | Recruiting ➔ Active, not recruiting

Enrollment closed • Gene therapy • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Long Term Follow-Up Gene Therapy Study for XLRP RPGR (clinicaltrials.gov) - P=N/A | N=55 | Active, not recruiting | Sponsor: Janssen Research & Development, LLC | N=42 ➔ 55

Enrollment change • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene (clinicaltrials.gov) - P3 | N=97 | Recruiting | Sponsor: Janssen Research & Development, LLC | Active, not recruiting ➔ Recruiting

Enrollment open • Gene therapy • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene (clinicaltrials.gov) - P3 | N=97 | Recruiting | Sponsor: Janssen Research & Development, LLC | Active, not recruiting ➔ Recruiting | Trial completion date: Sep 2024 ➔ Feb 2025

Enrollment open • Gene therapy • Trial completion date • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Long Term Follow-Up Gene Therapy Study for XLRP RPGR (clinicaltrials.gov) - P1/2 | N=42 | Active, not recruiting | Sponsor: MeiraGTx UK II Ltd | Recruiting ➔ Active, not recruiting | Trial completion date: Jun 2023 ➔ Nov 2026 | Trial primary completion date: Jun 2023 ➔ Nov 2026

Enrollment closed • Gene therapy • Trial completion date • Trial primary completion date • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene (clinicaltrials.gov) - P3 | N=97 | Active, not recruiting | Sponsor: MeiraGTx UK II Ltd | Recruiting ➔ Active, not recruiting | Trial completion date: Mar 2024 ➔ Sep 2024 | Trial primary completion date: Mar 2024 ➔ Sep 2024

Enrollment closed • Gene therapy • Trial completion date • Trial primary completion date • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene (clinicaltrials.gov) - P3 | N=97 | Active, not recruiting | Sponsor: MeiraGTx UK II Ltd | Recruiting ➔ Active, not recruiting | Trial completion date: May 2029 ➔ Sep 2029 | Trial primary completion date: May 2029 ➔ Sep 2029

Enrollment closed • Gene therapy • Trial completion date • Trial primary completion date • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Janssen to Highlight Innovation in Retinal Pipeline at the Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting (PRNewswire) - "The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that five company-sponsored presentations will be featured during the Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting in New Orleans from April 23-27, 2023. Janssen presentations will include updates from the Phase 1/2 MGT009 trial for investigational gene therapy botaretigene sparoparvovec (bota-vec, formerly AAV-RPGR) in patients with the inherited retinal disease (IRD) X-linked retinitis pigmentosa (XLRP) associated with the retinitis pigmentosa GTPase regulator (RPGR) gene, and pooled safety analysis data from two Phase 1 trials of the investigational gene therapy JNJ-81201887 (JNJ-1887); one trial in patients with geographic atrophy (GA), a late-stage and severe form of age-related macular degeneration (AMD) and one trial in wet AMD (Abstracts #5446 and #732-C0314)."

P1/2 data • P2 data • Age-related Macular Degeneration • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Humoral immune response to AAV5-RPGR (botaretigene sparoparvovec) gene therapy in RPGR-associated X-linked retinitis pigmentosa (ARVO 2023) - P1/2 | "Findings did not seem to deviate from those reported for voretigene neparvovec. Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

Gene therapy • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene (clinicaltrials.gov) - P3 | N=96 | Recruiting | Sponsor: MeiraGTx UK II Ltd | N=66 ➔ 96 | Trial completion date: Oct 2028 ➔ May 2029 | Trial primary completion date: Oct 2024 ➔ May 2029

Enrollment change • Trial completion date • Trial primary completion date • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene (clinicaltrials.gov) - P3 | N=96 | Recruiting | Sponsor: MeiraGTx UK II Ltd | N=66 ➔ 96 | Trial completion date: Oct 2023 ➔ Mar 2024 | Trial primary completion date: Oct 2023 ➔ Mar 2024

Enrollment change • Trial completion date • Trial primary completion date • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Ph1/2 AAV5-RPGR (Botaretigene Sparoparvovec) Gene Therapy Trial in RPGR-associated X-linked Retinitis Pigmentosa (XLRP) (AAO 2022) - No abstract available

Late-breaking abstract • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene (clinicaltrials.gov) - P3 | N=66 | Recruiting | Sponsor: MeiraGTx UK II Ltd | Trial completion date: Jul 2027 ➔ Oct 2028 | Trial primary completion date: Jul 2023 ➔ Oct 2024

Trial completion date • Trial primary completion date • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene (clinicaltrials.gov) - P3 | N=66 | Recruiting | Sponsor: MeiraGTx UK II Ltd | Trial completion date: Jul 2022 ➔ Oct 2023 | Trial primary completion date: Jul 2022 ➔ Oct 2023

Trial completion date • Trial primary completion date • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa