Gene therapies for rare eye diseases

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.