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 and NPHP1 both related LCA. VARIANT prepares a first-in-human clinical trial for ciliopathies in collaboration with “Institut IMAGINE” and “Hôpital Necker Enfants malades.
These discoveries open the door to expanding Variant’s therapeutic scope beyond monogenic diseases to more complex genetic syndromes