Gene Therapy

The investigational RNA therapy is designed to address the underlying cause of Leber congenital amaurosis 10 due to a mutation in the CEP290 gene.

ViGeneron and Biogen this week have announced a global collaboration and licensing agreement in which the partners will develop and commercialize gene therapy products based on adeno-associated virus (AAV) vectors that treat inherited eye disease.

Nine pediatric patients with Leber congenital amaurosis show visual improvement in clinical trials.

With several clinical trials underway, ocular therapy is seen as having potential in the treatment of a number of gene-related diseases.

Patients with pathogenic variants experience lower VA tied to length of IS/OS junction.

Investigators reviewed visual acuity, multi-modal retinal imaging, electroretinography, family history.

Study examines use of fluocinolone acetonide for treating DME in patients.

This ability would unlock possibilities for repairing tissue damaged by disease

Intravitreal gene therapy continues to be well tolerated and shows robust efficacy

In study, treatment demonstrates promising biologic activity, safety profile

Patients underwent short-course, perioperative systemic immunosuppression prior to procedure

Investigators find that nanoparticles deliver gene therapy successfully in mice, rats.

Investigators find that nanoparticles deliver gene therapy successfully in mice, rats.