University of Campania Luigi Vanvitelli
🇮🇹Italy
Variant Secures EMA Endorsement for VAR002 Gene Therapy Targeting Inherited Retinal Dystrophies
• The European Medicines Agency has provided positive scientific advice on Variant's VAR002, an adeno-associated viral vector gene therapy targeting inherited retinal dystrophies linked to CRX mutations.
• VAR002's non-clinical development plan was deemed suitable for advancing to first-in-human clinical trials, with the EMA confirming that planned pivotal toxicology studies align with requirements for future EU marketing authorization.
• The gene replacement therapy shows potential beyond CRX-related disorders, with preclinical studies demonstrating efficacy in diseases associated with phototransduction pathway defects and ciliopathies, including CEP290-associated conditions.
AAVantgarde Bio Doses First Patient in Phase 1/2 Trial of AAVB-081 for Usher Syndrome Type 1B
• AAVantgarde Bio has dosed the first patient in its Phase 1/2 LUCE-1 trial, evaluating AAVB-081 gene therapy for Usher Syndrome Type 1B (USH1B).
• AAVB-081 is a dual hybrid AAV8 vector designed to deliver a functional copy of the Myo7A gene via subretinal administration.
• The open-label, dose-escalation LUCE-1 trial will enroll 15 patients aged 18-50 with USH1B, assessing safety and efficacy endpoints over 61 months.
• Researchers are also exploring gene-editing approaches for other forms of Usher syndrome, such as Usher syndrome type 2A (USH2A), to address hearing and vision loss.
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