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

PARIS – Variant, a company dedicated to developing innovative treatments for inherited eye orphan diseases, 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, comments, “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 !”.


VAR002 is a recombinant adeno-associated viral (rAAV) vector developed to treat LCA, CORD and RP due to a mutation in the CRX gene. This gene replacement therapy provides an unmutated copy of the human CRX gene to replace the defective gene, inducing the expression of a functional CRX protein in the photoreceptors. VAR002 is administered as a sterile suspension of viral particles, injected directly into the subretinal space. This triggers the expression of the transgene in the rods and the cones.


Variant is a biotechnology company that develops innovative products in the ophthalmology sector. Variant aims to provide the pharmaceutical industry with solid proof-of-concept data to establish therapeutic strategies for rare visual diseases. The company has several scientific collaborations specialized in antisense oligonucleotides (ASO)°, genome analysis, production and administration of AAV vectors, and human retinal organoids. Our associated partners are well-positioned to test therapies for dominant and recessive retinal dystrophy forms, notably orphan diseases, such as congenital Leber Amaurosis, cone-rod dystrophies, and retinitis pigmentosa. Variant’s leading preclinical portfolio has a diversified risk profile: the company specializes in using “transcription factor”, which intervene very early in the development of photoreceptors to relaunch the differentiation of cones and rods in the retina. The company achieved an exciting combination of therapeutic approaches. On one side, for moderate CRX CORD a less invasive intravitreal administration of encouraging ASO treatment. And, on the other side, the experimented subretinal administration of AAV vector for severe CRX LCA & RP.

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Media : FP2COM
Florence Portejoie
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