First patient dosed in ViGeneron Phase 1b clinical trial of VG901 for the intravitreal treatment of RP

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ViGeneron GmbH announced the first patient has been dosed in its Phase 1b clinical trial (NCT06291935) evaluating intravitreal injection of VG901 to treat retinitis pigmentosa (RP) caused by mutations in the CNGA1 gene.

According to the company, the milestone marks an important advance as the company continues to leverage its next-generation technology platforms to develop groundbreaking gene therapies addressing critical unmet medical needs.

Clinical trial participants will be administered a single intravitreal dose of VG901 into the most affected eye through a syringe and followed up for a year to monitor safety and efficacy. There will be two cohorts of participants in this study. Study Cohort 1 will receive the low dose and Study Cohort 2 will receive the high dose as specified in the protocol.¹

The study is expected to be completed by the end of 2025.¹

Katarina Stingl, MD, noted in a news release that delivering a functional CNGA1 gene to retinal photoreceptor target cells, VG901 offers a therapeutic potential in addressing the genetic root cause for patients with retinitis pigmentosa affected by CNGA1 mutations.²

Stingl is the head of the Clinic for Hereditary Retinal Degenerations in the Center for Ophthalmology and the Center for Rare Eye Diseases at the University of Tübingen, Germany, and the principal investigator for this trial. "We are excited to learn about the potential of this novel therapy and hope to make a meaningful difference to patients' lives,” said Stingl.

Caroline Man Xu, PHD, co-founder and CEO of ViGeneron, pointed out in the news release that dosing the first patient in the VG901 Phase Ib clinical trial is a step forward for the company and for the patients.

“VG901 has also been granted FDA Orphan Drug Designation status. We look forward to progressing the clinical development of this potentially transformative therapy,” she said in the news release. “Not only is the Phase Ib trial designed to provide key insights into the safety and preliminary efficacy of VG901, it is also a pivotal step in validating our next generation vector platform vgAAV, which has demonstrated superior transduction efficiency and enables intravitreal delivery.”

According to the company, the on-going Phase 1b clinical trial is an open-label, single-arm, dose-escalation study investigating the safety, tolerability, and preliminary efficacy of an intravitreal injection of VG901, a first-in-class CNGA1 gene therapy for autosomal recessive RP.

Reference:

1. ViGeneron GmbH. A Prospective, Open-label, Phase 1b, Single-arm, Safety Study of an Intravitreal Application of a Recombinant Adeno-associated Virus Vector Expressing CNGA1 (AAV2.NN-CNGA1) in Patients With Retinitis Pigmentosa Due to CNGA1 Mutations. clinicaltrials.gov. Published February 26, 2024. Accessed April 10, 2024. https://classic.clinicaltrials.gov/ct2/show/study/NCT06291935

2. ‌ViGeneron Announces First Patient Dosed in Phase 1b Clinical Trial of VG901 for the Intravitreal Treatment of Retinitis Pigmentosa. Yahoo Finance. Published April 10, 2024. Accessed April 10, 2024. https://finance.yahoo.com/news/vigeneron-announces-first-patient-dosed-090300678.html