Gene therapies for rare eye diseases

A fight against blindness!

News & Press releases

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 Medicines Agency (EMA) on VAR002, an adeno-associated viral vector gene therapy targeting inherited retinal dystrophies linked to CRX mutations. This consultation and subsequent feedback from…

VARIANT reçoit un avis favorable de l’Agence Européenne des Médicaments (EMA) pour VAR002, sa thérapie génique de remplacement innovante.

Apr 1, 2025

Paris – Variant et le Centre des Maladies Oculaires Rares de l’Université de Campanie Luigi Vanvitelli – UCLV (Naples, Italie), annoncent aujourd’hui avoir reçu un avis favorable de l’EMA. Cette consultation visait à garantir que le plan de développement non clinique de VAR002 est adapté à la réalisation d’un futur essai clinique de première administration…

VARIANT and University of Campania Luigi Vanvitelli partner on pre-clinical studies for VAR002, a gene therapy for CRX-related Inherited retinal diseases.

May 5, 2024

Paris – Variant and the Rare Ocular Diseases Center at the University of Campania Luigi Vanvitelli – UCLV (Naples, Italy) today announce a collaboration to initiate pre-clinical studies to accelerate the development of VAR002. These studies include a Natural History Study in patients with CRX-related retinal dystrophies, GLP/GMP vector production and a pre-clinical toxicological study…

Denis-CAYET-Tedx

Denis CAYET
CEO

apos1

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.

apos2

Our history

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.

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.

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

apos1

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!

apos2

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