ZVS101e / Zhongyin Technology - 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

A 30-year-old man with Bietti crystalline dystrophy:a rare case report from Syria. (PubMed, Ann Med Surg (Lond)) - "Subretinal rAAV2/8-hCYP4V2 gene therapy for BCD is safe and effective, but COVID-19 or AAV8 antibodies may hinder its efficacy...Health care professionals should be vigilant in recognizing rare eye diseases like BCD, even in uncommon regions. Further research is crucial to understand BCD, develop treatments, and improve the quality of life for affected individuals."

Journal • Dyslipidemia • Gene Therapies • Hypertriglyceridemia • Infectious Disease • Inherited Retinal Dystrophy • Metabolic Disorders • Novel Coronavirus Disease • Ophthalmology

Phase 1/2 Gene Therapy Trial for Bietti Crystalline Corneoretinal Dystrophy (ARVO 2025) - "Based on the outstanding efficacy of ZVS101e, it has received regenerative medicine advanced therapies (RMAT) designation from the US FDA and breakthrough therapy (BTD) designation from the National Medicinal Products Administration (NMPA) of China. Phase 3 clinical trial is currently underway."

Gene therapy • P1/2 data • Inherited Retinal Dystrophy • Ophthalmology

Efficacy and Safety of ZVS101e in Patients With Bietti 's Crystalline Dystrophy (clinicaltrials.gov) - P3 | N=62 | Recruiting | Sponsor: Chigenovo Co., Ltd

New P3 trial • Inherited Retinal Dystrophy • Ophthalmology

Safety and Efficacy of ZVS101e in Patients With Bietti 's Crystalline Dystrophy (clinicaltrials.gov) - P1/2 | N=24 | Active, not recruiting | Sponsor: Chigenovo Co., Ltd | Recruiting ➔ Active, not recruiting | Trial primary completion date: Dec 2028 ➔ Dec 2024

Enrollment closed • Trial primary completion date

Gene replacement therapy in Bietti crystalline corneoretinal dystrophy: an open-label, single-arm, exploratory trial. (PubMed, Signal Transduct Target Ther) - P1 | "Importantly, the patients' improvement observed using multifocal electroretinogram, microperimetry, and Visual Function Questionnaire-25 further supported the beneficial effects of the treatment. We conclude that the favorable safety profile and visual improvements identified in this trial encourage the continued development of rAAV2/8-hCYP4V2 (named ZVS101e)."

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

Safety and Efficacy of AAV-mediated Gene Replacement Therapy in Bietti Crystalline Corneoretinal Dystrophy (ARVO 2024) - P1 | "We conclude that the favorable safety profile and visual improvements identified in this trial encourage the continuation of rAAV2/8-hCYP4V2 (ZVS101e) development."

Clinical • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

Safety and Efficacy of ZVS101e in Patients With Bietti 's Crystalline Dystrophy (clinicaltrials.gov) - P1/2 | N=24 | Recruiting | Sponsor: Chigenovo Co., Ltd

New P1/2 trial

Safety and Efficacy of AAV-Mediated Gene Replacement Therapy in Bietti Crystalline Corneoretinal Dystrophy (BCD) Patients with CYP4V2 mutations (ARVO 2023) - P1 | "All subjects received recombinant adeno-associated virus that contains the gene for human CYP4V2 protein (rAAV2/8-hCYP4V2) by single subretinal administration with 150µL volume of drug at a dose of 7.5×10 10 viral genomes (vg)...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."

Clinical • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

Safety Study of rAAV2/8-hCYP4V2 in Patients With Bietti's Crystalline Dystrophy (BCD) (clinicaltrials.gov) - P1 | N=12 | Recruiting | Sponsor: Beijing Tongren Hospital | Trial completion date: Dec 2023 ➔ Apr 2024 | Trial primary completion date: Jun 2023 ➔ Jan 2024

Trial completion date • Trial primary completion date

Safety and Tolerability of ZVS101e in Patients With Bietti 's Crystalline Dystrophy (clinicaltrials.gov) - P1 | N=6 | Recruiting | Sponsor: Peking University Third Hospital

New P1 trial