Gene therapy specialist Shanghai BDgene Technology Co., Ltd has announced the conclusion of three clinical research treatments using its HELP technology, based on the CRISPR-Cas9 gene editing tool, for herpes simplex keratitis (HSK). Preliminary positive results have been published in the journal Molecular Therapy, in an article titled “In Vivo CRISPR Gene Editing in Patients with Herpes Stromal Keratitis” (HSK).
Understanding Herpes Stromal Keratitis (HSK)
HSK is a leading cause of vision loss and potential blindness following herpes simplex virus (HSV)-1 infection. The condition results in corneal scarring and neovascularization due to inflammation. In the infected cornea, HSV can activate the innate and adaptive immune responses of host cells, leading to the release of inflammatory cytokines, chemokines, microRNA, and other regulatory factors that can either stimulate or inhibit tissue responses.
Clinical Study Results and Implications
In the clinical study (NCT04560790), three patients with severe refractory HSK and acute corneal perforation were treated with HELP in combination with corneal transplantation, with an average follow-up period of 18 months. The results demonstrated no recurrence of HSV-1 in the patients, no off-target effects induced by CRISPR, and no systemic adverse events, indicating the potential safety and efficacy of the treatment.-Fineline Info & Tech
