VARIANT strengthens its versatile gene therapy platform with a new patent targeting retinal ciliopathies

Paris, November 28, 2025 – VARIANT, a French company specializing in gene therapies for rare retinal diseases, announces the filing of a new patent protecting the use of the transcription factor CRX to treat retinal ciliopathies, in partnership with CNRS (Université Paris-Saclay) and the Institut Imagine (INSERM, AP‑HP, Université Paris Cité).

Ciliopathies include, among others, Leber congenital amaurosis, retinitis pigmentosa, cone‑rod dystrophy, as well as certain syndromes such as Bardet‑Biedl and Joubert. Together, these rare diseases represent nearly one quarter of hereditary retinal dystrophies still without treatment — an area in which VARIANT aims to pave the way for new therapeutic options through its gene therapy platform.

A new patent on the use of the transcription factor CRX

Together with CNRS (Université Paris-Saclay) and the Institut Imagine (INSERM, AP‑HP, Université Paris Cité), VARIANT is expanding the scope of its product VAR‑002 with a patent protecting the use of the transcription factor CRX for the treatment of retinal ciliopathies, a group of rare genetic diseases affecting the structure and function of photoreceptor cilia.

This patent is based on an innovative approach designed to restore the coordinated expression of genes essential for phototransduction and ciliary integrity, independent of the originally mutated gene. By leveraging the central role of CRX as the master regulator of the photoreceptor genetic program, this strategy opens the door to therapeutic applications capable of targeting several forms of retinal dystrophies that currently have no treatment.

The patent is supported by results from preclinical studies conducted on animal models reproducing different ciliopathies: a long‑term study on CEP290 models, carried out at CERTO (Centre d’Évaluation et de Recherche en Thérapie Oculaire – CNRS), which confirmed the durability and safety of CRX expression in photoreceptor cells; and a pilot study conducted by the Institut Imagine on NPHP1 models, demonstrating partial functional restoration of the retina and significant improvement of morphological markers.

These studies strengthen the relevance of the CRX approach as a “multi‑target” gene therapy for retinal ciliopathies, further establishing VARIANT’s position among Europe’s pioneers in ocular genetic medicine.

About VAR-002

VAR‑002 is a next‑generation gene therapy developed by VARIANT to treat hereditary retinal dystrophies, whether or not a causal mutation has been identified. This treatment relies on the transcription factor CRX, likely the most powerful natural regulator of the photoreceptor genetic program — the key visual cells known as cones and rods.

Unlike traditional approaches that replace a defective gene with a healthy copy, VAR‑002 works by reactivating the gene networks governed by CRX to restore visual function. By delivering CRX directly to photoreceptors using an adeno‑associated viral vector (rAAV) specifically active in these cells, VAR‑002 promotes neuronal survival, compensates for partial loss of function of altered proteins, and reinitiates the differentiation of dormant cones. This gene‑agnostic strategy makes it possible to target both CRX‑related diseases and many other dystrophies, including those involving CEP290, NPHP1, or ABCA4, thereby offering a cross‑cutting solution for conditions that previously lacked treatment.

About VARIANT

VARIANT is a French biotechnology company specializing in the development of innovative gene therapy solutions for hereditary retinal diseases. The company focuses on leveraging retinal transcription factors to restore photoreceptor function in severe retinal dystrophies.

VARIANT is currently preparing the preclinical regulatory phase of its lead candidate, VAR‑002, with the launch of a toxicology study scheduled for 2026 — a key step before entering clinical trials. Two phase I/II clinical trials are already in preparation: the first, conducted in collaboration with the Center for Rare Ocular Diseases at the University of Campania Luigi Vanvitelli in Naples, will focus on hereditary retinal dystrophies caused by CRX mutations; the second, conducted with the ophthalmology department of Necker–Enfants Malades Hospital (AP‑HP) in Paris, will target retinal ciliopathies, a group of rare diseases for which no treatment is currently available.

About Institut Imagine

As Europe’s leading center for research, care, and education on genetic diseases, the Imagine Institute brings together nearly 1,000 professionals on the Necker–Enfants Malades hospital in Paris. Through its 31 affiliated rare disease reference centers, the Institute unites patients, families, physicians, and researchers under one roof to accelerate discoveries in genetic disorders — the leading cause of infant mortality in France.

About CNRS

A major global player in fundamental research, the Centre national de la recherche scientifique (CNRS) is the only research organization in France active across all scientific fields. Its uniquely broad expertise enables the CNRS to combine multiple disciplines to shed light on, and address the challenges facing today’s world, in collaboration with public and socio-economic stakeholders. Together, all scientific disciplines are mobilized in the service of sustainable progress that benefits society as a whole

About CERTO

CERTO is a private laboratory of the Retina France association, integrated within l’Institut des neurosciences Paris-Saclay (NeuroPSI – CNRS/Université Paris-Saclay). In addition to its internal research programs, CERTO develops research partnerships with academic laboratories and biotechnology companies.

About Retina France

Founded in 1984, Retina France is a non-profit patient association recognized as being of public interest since November 6, 1998. Its missions are to: 1) Bring together individuals affected by visual disorders to inform them and provide social and humanitarian assistance, helping patients and their families overcome the challenges associated with visual impairment. 2) Promote, encourage, and support therapeutic research. 3) More broadly, undertake any actions necessary to inform and engage the public and patients about the Association’s goals, as well as the results achieved by the Association and by scientific researchers.