Variant Confirms Breakthrough in Treatment of Hereditary CRX-Dependent Retinopathies with an Innovative Gene Therapy Product VAR002

VAR002 is protected by an international patent application which claims this innovative product of gene therapy in its therapeutic applications until 2040, primarily for autosomal dominant inherited retinal dystrophies associated with mutations in the CRX transcription factor.

Paris, France, March 30, 2021, 6 p.m. – Variant, a company dedicated to developing innovative treatments for inherited eye orphan diseases. confirms its position as a major player in the field of the treatment of retinopathies with its product VAR002 – an innovative gene therapy based on AAV vectors – by the publication of its international patent application published under number WO2020 / 182722.

This patent will protect this new gene therapy product for primarily treating patients with dominant CRX mutations, genetic alterations leading to a group of progressive retinal dystrophies such as Leber congenital amaurosis (LCA), cone-rod dystrophy (CORD) and retinitis pigmentosa (RP).

“We are ready to start a toxicology study followed by a phase I/II clinical trial after the first signs of preclinical efficacy demonstrated in different in vivo and in vitro models. VAR002 has strong developmental potential to meet the needs of patients carrying dominant CRX mutations”, comments Jerome ROGER, project manager. Our objective is to overexpress wild-type CRX in photoreceptors to compensate for the deleterious effect of the mutated form of CRX. Our strategy should target a vast majority of CRX mutations, independently of the associated pathology as LCA, CORD or RP. The positive results of the preclinical studies of VAR002 showed efficacy and a good safety profile at the defined dose. These studies determined the optimal dose for the future toxicology study in view of a phase1/2 clinical trial.

Denis CAYET, Variant CEO, comments “We are now in discussions to move forward in the clinic. If we quickly find a strong partner used to deal with clinical trials in ophthalmology, we should be able to bring a nice new solution to many patients”.


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 enable the clinical development of therapeutic strategies intended for rare visual diseases. The company has several scientific collaborations specialized in 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 the use of “transcription factor” genes, which intervene very early in the development of photoreceptors to relaunch the differentiation of cones and rods in the retina.


Media : FP2COM
Florence Portejoie
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