VARIANT and University of Campania Luigi Vanvitelli partner on pre-clinical studies for VAR002, a gene therapy for CRX-related Inherited retinal diseases.
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 in large size-animal models.
Variant and UCLV Partner on Pre-Clinical Development of CRX Gene Therapy
To ensure success in upcoming clinical trials for VAR002, gene therapy for CRX-related inherited retinal diseases (Leber congenital amaurosis, Retinitis pigmentosa, and Cone-rod dystrophy), Variant is collaborating with the Centre for Rare Ocular Diseases of University of Campania Luigi Vanvitelli (UCLV).
Understanding CRX Disease Progression is Key
This collaboration focuses on two crucial aspects:
- Natural History Study: Led by Prof. Dr Francesca Simonelli, Chief of the Eye Clinic at UCLV, a longitudinal study will follow CRX patients for at least two years. This will help establish optimal clinical endpoints and testing methods to measure disease progression.
- Pre-Clinical Development: VARIANT will conduct a pre-clinical toxicology study and vector production with UCLV. This ensures the safety of VAR002 before clinical trials begin in Naples.
Securing the Path to a CRX Therapy
“The Centre for Rare Ocular Diseases at UCLV has extensive international experience in ophthalmological gene therapy clinical trials,” says Denis Cayet, CEO of Variant. “This collaboration allows us to leverage their expertise for pre-clinical development, ultimately paving the way for the first CRX gene therapy clinical trial in Naples.”
A chance for CRX patients
Prof. Dr. Simonelli adds, “This natural history study will provide valuable insights into progression of CRX-related diseases, allowing us to develop better outcome measures. With VAR002, we believe that CRX patients will have a promising new treatment option.”
ABOUT VAR002
VAR002 is a recombinant adeno-associated viral (rAAV) vector developed to treat Leber’s congenital amaurosis, Retinitis pigmentosa, and Cone-rod dystrophy due to a mutation in the CRX gene. This gene replacement therapy provides an unmutated copy of the human CRX gene to replace the defective gene, inducing the expression of a functional CRX protein in the photoreceptors. VAR002 is administered as a sterile suspension of viral particles, injected directly into the subretinal space. This triggers the expression of the transgene in the rods and the cones.
ABOUT VARIANT
Variant is a biotechnology company that develops innovative products in the ophthalmology sector. Variant aims to provide the pharmaceutical industry with solid proof-of-concept data to enable the clinical development of therapeutic strategies for rare visual diseases. The company has several scientific collaborations in genome analysis, production and administration of AAV vectors, and human retinal organoids. Our associated partners are well-positioned to test therapies for dominant and recessive retinal dystrophy forms, notably orphan diseases, such as Leber Congenital Amaurosis, cone-rod dystrophies, and retinitis pigmentosa. In addition, Variant’s leading pre-clinical portfolio has a diversified risk profile: the company specializes in using “transcription factor” genes, which intervene very early in developing photoreceptors to relaunch the differentiation of cones and rods in the retina.
ABOUT The Centre for Rare Ocular Diseases Center at University of Campania Luigi Vanvitelli, Naples, Italy.
The Centre for Rare Ocular Diseases of the academic hospital University of Campania Luigi Vanvitelli offers diagnostic, rehabilitative and therapeutic courses for patients suffering from hereditary eye diseases, such as inherited retinal dystrophies, hereditary optic diseases, keratoconus and corneal dystrophies, both in non-syndromic and syndromic form. Recently, a “Telethon Advanced Ocular Therapy Unit” was created at the Centre, born from the synergy between the Telethon Foundation and the academic hospital University of Campania Luigi Vanvitelli, unique in Italy and among the few in Europe, dedicated to clinical studies aimed to evaluate innovative therapeutic strategies for the treatment of rare ocular diseases. This way, patients can be enrolled in major clinical studies for hereditary eye diseases.