GenEditBio has dosed the first subject in an investigator-initiated trial of its in vivo genome editing programme, GEB-101, for transforming growth factor beta induced (TGFBI) corneal dystrophy.
The dose-escalation and open-label clinical trial, conducted in partnership with the Eye & ENT Hospital of Fudan University in Shanghai, China, aims to assess the tolerability of the programme when used in conjunction with standard phototherapeutic keratectomy treatment in the adult population with this condition.
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The first subject who was treated with the therapy was discharged without any adverse events being observed.
According to the company, TGFBI corneal dystrophy is a genetic disorder that leads to the accumulation of abnormal proteins in the cornea’s stromal layer, with symptoms being loss of vision, photophobia, as well as recurrent corneal erosions.
While treatments such as corneal transplantation and phototherapeutic keratectomy are available, they have limitations and risks.
GEB-101 is designed as a once-and-done treatment that leverages clustered regularly interspaced short palindromic repeats (CRISPR)-Cas genome editing technology to target a specific mutation in the TGFBI gene.
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By GlobalDataEncapsulated in an engineered protein delivery vehicle (PDV) for delivery, the programme is administered via intrastromal injection and is currently under evaluation for its tolerability in this trial.
GenEditBio co-founder and chairman Zongli Zheng said: “We stand at the frontier of a new era and recognise that the transformative potential of this moment is not just for the company but for the entire field of genetic medicine because the technology has the potential to extend far beyond corneal dystrophy.
“Our company is committed to developing fundamentally safe, efficacious and affordable in vivo genome editing therapies for genetic diseases with unmet medical needs.”
Established in 2021 and headquartered in China, the startup specialises in gene therapy, focusing on Cas nuclease discovery and the development of safe cargo delivery systems using lipid nanoparticles and PDVs.
