AAV-CNGA3 / J&J, MeiraGTx - LARVOL DELTA

Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and CNGA3 (clinicaltrials.gov) - P1/2 | N=34 | Completed | Sponsor: MeiraGTx UK II Ltd | Active, not recruiting ➔ Completed | Trial completion date: Jan 2026 ➔ Apr 2024 | Trial primary completion date: Jan 2026 ➔ Apr 2024

Gene therapy • Trial completion • Trial completion date • Trial primary completion date • Achromatopsia • Gene Therapies • Ophthalmology

A Bioengineered In Vitro Model to Assess AAV-Based Gene Therapies for Cyclic GMP-Related Disorders. (PubMed, Int J Mol Sci) - "Our system is based on an immortalized cell line with high susceptibility to AAV transduction, i.e., HeLa cells, which we engineered to express a fungal rhodopsin guanylyl cyclase (RhGC) from Blastocladiella emersonii and a sensitive genetically encoded calcium indicator (GECI) under the control of a tetracycline operator. Using this system, we were able to confirm and quantify the function of the ion channel encoded by AAV/CNGA3 and differentiate between AAV vector potencies with a simple fluorometric assay. Finally, we show that this approach can be readily adapted for the assessment of phosphodiesterase function."

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

Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and CNGA3 (clinicaltrials.gov) - P1/2; N=34; Active, not recruiting; Sponsor: MeiraGTx UK II Ltd; Recruiting ➔ Active, not recruiting; N=72 ➔ 34; Trial completion date: Aug 2024 ➔ Jan 2026; Trial primary completion date: Jul 2024 ➔ Jan 2026

Clinical • Enrollment change • Enrollment closed • Trial completion date • Trial primary completion date • Achromatopsia • Gene Therapies • Ophthalmology

Gene Therapy for Achromatopsia (CNGA3) (clinicaltrials.gov) - P1/2; N=11; Completed; Sponsor: MeiraGTx UK II Ltd; Recruiting ➔ Completed; N=18 ➔ 11; Trial completion date: Jan 2022 ➔ Jun 2021

Clinical • Enrollment change • Trial completion • Trial completion date • Achromatopsia • Gene Therapies • Ophthalmology