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Gene therapies for rare eye diseases

News & Press releases

VARIANT renforce sa plateforme de thérapie génique « polyvalente » avec un nouveau brevet ciblant les ciliopathies rétiniennes  

Nov 28, 2025

Paris, le 28 novembre 2025 – VARIANT, société française spécialisée dans les thérapies géniques pour les maladies rares de la rétine, annonce le dépôt d’un nouveau brevet protégeant l’utilisation du…

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

Nov 28, 2025

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…

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VARIANT Receives Positive Feedback from the European Medicines Agency(EMA) for its Innovative Gene Replacement Therapy, VAR002

Apr 1, 2025

PARIS – Variant and the Rare Ocular Diseases Center at the University of Campania Luigi Vanvitelli (UCLV) in Naples today announced that they have received positive feedback from the European…

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Denis-CAYET-Tedx

Denis CAYET, CEO

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Our research team has demonstrated and patented the therapeutic potential of transcription factors, paving the way for a broader spectrum of treatments for inherited retinal diseases.

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Our history

01

Vision Driven by Science—and by Experience

Variant was founded by Denis Cayet, who was himself diagnosed with a dominant form of macular dystrophy. His personal experience catalyzed the creation of a biotech company with a singular goal: finding curative treatments for retinal degenerations.

02

Scientific Leadership and Pipeline Origins

In 2017, Denis assembled a dedicated research team to establish proof-of-concept and secure intellectual property. The team included:

  • Thomas Lamonnerie, expert in retinal transcription factors, who pioneered a novel antisense oligonucleotide (ASO) inhibition strategy (VAR003, currently in discovery).
  • Jérôme Roger, a specialist in ophthalmic gene therapy, who initiated an expression-based approach using CRX, a master gene in photoreceptor development.

Less than two years later, VAR002, based on CRX gene delivery, showed strong efficacy in preclinical models of CRX-linked Leber Congenital Amaurosis.

03

Toward the Clinic—and Beyond

In 2024, Variant established a Translational Preclinical Task Force to prepare a first-in-human clinical trial in collaboration with Prof. Francesca Simonelli (Naples, Italy), targeting CRX-associated pathologies.

That same year, new research revealed that VAR002 may benefit a broader spectrum of IRDs, including certain ciliopathies such as CEP290-related LCA.

These discoveries open the door to expanding Variant’s therapeutic scope beyond monogenic diseases to more complex genetic syndromes.

Denis-CAYET-Tedx

Denis CAYET, CEO

The safe and powerful CRX master gene, encapsulated in VAR002, is now ready for multiple clinical trials targeting key inherited retinal diseases. A new step forward in gene therapy for vision restoration!

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