VGR-R01 / Beijing Tongren Hospital - LARVOL DELTA

Bietti crystalline corneoretinal dystrophy: Advances in understanding and gene therapeutic approaches. (PubMed, Mol Vis) - "We specifically analyze the clinical outcomes of three investigational gene therapy products-ZVS101e, NGGT001, and VGR-R01-focusing on their preliminary efficacy, safety profiles, and tolerability. Future optimization of these approaches should carefully consider administration sites and modalities, injection volumes, and disease severity at intervention. With gene replacement therapy for BCD advancing through late-stage clinical development, regulatory approval and clinical implementation may be anticipated in the near future."

Journal • Review • Gene Therapies • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

Preclinical studies of an AAV8-CYP4V2 gene therapy VGR-R01 for the treatment of Bietti crystalline dystrophy. (PubMed, Mol Ther Methods Clin Dev) - P1 | "Furthermore, after subretinal administration, VGR-R01 was primarily distributed in the ocular tissues, especially in the retina, with minimal systemic presence, notably in the gonads. Overall, these results support the potential for clinical application of VGR-R01 in treating BCD."

Journal • Preclinical • Gene Therapies • Inherited Retinal Dystrophy • Ophthalmology

Study to Evaluate the Efficacy and Safety of VGR-R01 Gene Therapy in Patients with Bietti Crystalline Dystrophy (clinicaltrials.gov) - P3 | N=45 | Not yet recruiting | Sponsor: Shanghai Vitalgen BioPharma Co., Ltd.

Gene therapy • New P3 trial • Gene Therapies • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

Subretinal Gene Therapy VGR-R01 for Bietti Crystalline Dystrophy (AAO 2024) - P1 | "Four subjects had ocular inflammation associated with VGR-R01 and inconsequential to the visual acuity. Conclusion VGR-R01 is well tolerated, with favorable safety profile, and efficacy is observed."

Gene therapy • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology

Subretinal Gene Therapy in Patients with Bietti Crystalline Dystrophy: Preliminary Results of a Phase 1/2 Clinical Trial (ASGCT 2024) - P1 | "VGR-R01 is safe and well tolerated, and efficacy is observed. More importantly, for some subjects with extremely low vision, the VGR-R01 administration also significantly improved their visual function.Plain Language Summary: Bietti crystalline dystrophy (BCD) caused by CYP4V2 biallelic mutation is a severe and visually threatening genetic disorder with no effective treatment. Here, we report the preliminary results of BCD patients received a single unilateral subretinal injection of VGR-R01, an AAV8 mediated CYP4V2 gene therapy."

Clinical • Gene therapy • P1/2 data • Gene Therapies • Inflammation • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology

Discovery and Characterization of a Safe and Efficacious Gene Replacement Therapy for Treating Bietti Crystalline Dystrophy (ASGCT 2023) - "In summary, VGR-R01, a safe and efficacious AAV-based gene therapy for treating BCD has been developed. Encouraging results from preliminary investigator-initiated-trials (IITs) have been obtained with Phase I/II trial patient enrollments ongoing in China."

Cardiovascular • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Metabolic Disorders • Ophthalmology

Initial Findings of Retinal Gene Therapy in Patients with Bietti Crystalline Dystrophy (ASGCT 2023) - P1 | "Subretinal gene therapy with VGR-R01 demonstrated a good safety profile and resulted in rapid improvement in functional vision in all participants with BCD. With more data being collected, we will continue to assess the effect of VGR-R01. A separate multicenter, open-label phase 1 study of VGR-R01 in patients with BCD is ongoing (NCT05694598)."

Clinical • Gene therapy • Gene Therapies • Inherited Retinal Dystrophy • Ophthalmology

Safety and Tolerability of VGR-R01 for Patients With Bietti Crystalline Dystrophy (clinicaltrials.gov) - P1 | N=12 | Not yet recruiting | Sponsor: Shanghai Vitalgen BioPharma Co., Ltd.

New P1 trial • Inherited Retinal Dystrophy • Ophthalmology

Safety and Tolerability of VGR-R01 in Patients With Bietti Crystalline Dystrophy (clinicaltrials.gov) - P1 | N=3 | Recruiting | Sponsor: Beijing Tongren Hospital | Not yet recruiting ➔ Recruiting | Initiation date: May 2022 ➔ Sep 2022

Enrollment open • Trial initiation date • Inherited Retinal Dystrophy • Ophthalmology

Safety and Tolerability of VGR-R01 in Patients With Bietti Crystalline Dystrophy (clinicaltrials.gov) - P1 | N=3 | Not yet recruiting | Sponsor: Beijing Tongren Hospital

New P1 trial • Inherited Retinal Dystrophy • Ophthalmology