Variant Receives European Orphan Drug Designation for its Gene Therapy Product Candidate, VAR002, for the Treatment of Both Leber Congenital Amaurosis and Cone-Rod dystrophy

Variant announces the decision of the European Commission to grant Orphan Drug Designation (ODD) for VAR002 for the treatment of Leber congenital amaurosis (LCA) and Cone-rod dystrophy (CORD). The decision is based on a positive opinion from the European Medicine Agency’s (EMA) Committee for Orphan Medicinal Products (COMP).

VAR002 is a CRX-expressing AAV vector designed as a relevant therapeutic approach for gene supplementation and a promising drug candidate for mutation-agnostic gene therapy. VAR002 prevents photoreceptor degeneration without adverse effects linked with CRX overexpression.

The ODD is an essential landmark in the development of VAR002 that, along with the strong intellectual property moving on to the regional phase (EU, US, and Japan), consolidates the position of VAR002 as CRX worldwide leader compared to the few other potential treatments involving transcription factors.

“With VAR002, I have the opportunity to approach the unmet needs of patients carrying dominant CRX mutations. We are excited to move this program to the clinic in collaboration with a new team experienced with translational development”, comments Jerome Roger, project manager. “Our strategy will target LCA, CORD, or RP associated with CRX mutations and many not CRX-related IRDs where the neuroprotective capacity had been proved. The positive results of the preclinical studies of VAR002 showed efficacy and a good safety profile at the defined dose”.

Denis Cayet, Variant CEO, added, “With the VAR002 ODDs and patent, VARIANT develops solid protection of its know-how on retinal transcription factors. As a result, we can move forward to bring an excellent new solution to CRX patients! And this future perspective makes me happy!”.