Variant, a France-based company involved in developing innovative treatments for orphan inherited eye diseases, and the Rare Ocular Diseases Centre at the University of Campania Luigi Vanvitelli (UCLV) in Naples, announced on Thursday that they have received positive feedback from the European Medicines Agency (EMA) for VAR002, an adeno-associated viral vector gene therapy targeting inherited retinal dystrophies linked to CRX mutations.

This consultation and subsequent feedback from the EMA ensure that VAR002's non-clinical development plan is suitable for a future first-in-human clinical trial. It also confirmed that the planned pivotal toxicology study design will support a Marketing Authorisation Application in the EU. The EMA provided scientific advice supporting the non-clinical development plan of VAR002, an adeno-associated viral vector gene therapy targeting inherited retinal dystrophies linked to CRX mutations.

The preclinical programme, including pivotal toxicology and biodistribution studies, was deemed appropriate to advance towards a first-in-human clinical trial. This endorsement confirms that the data generated so far adequately supports the safety and pharmacological profile of VAR002, ensuring a solid foundation for clinical translation.

The planned study will assess VAR002 in patients with CRX-associated retinal diseases, addressing a critical unmet medical need in ophthalmic gene therapy.