GenEditBio Begins Clinical Trial of GEB-101 for Corneal Dystrophy

GenEditBio Limited has announced the successful dosing of the first patient in an investigator-initiated trial (IIT) of GEB-101, the company’s novel in vivo genome editing therapy targeting TGFBI corneal dystrophy. This trial marks a significant milestone in the development of genetic treatments for corneal diseases.

TGFBI Corneal Dystrophy: A Genetic Disorder with Limited Options

TGFBI corneal dystrophy refers to a group of inherited eye disorders caused by mutations in the TGFBI gene, leading to abnormal protein accumulation in the stromal layer of the cornea. Current treatments are limited to phototherapeutic keratectomy (PTK) and corneal transplantation, which are primarily palliative and not curative.

Overview of GEB-101 and Its Mechanism of Action

GEB-101 is based on CRISPR-Cas genome editing technology, designed to directly target and edit the disease-causing mutation within the TGFBI gene. The therapeutic is formulated as a ribonucleoprotein (RNP) and delivered using GenEditBio’s proprietary protein delivery vehicle (PDV)—a specialized in vivo delivery platform engineered to enhance safety, precision, and localization.

In preclinical studies involving non-human primates, GEB-101 demonstrated strong safety outcomes. The therapy was well-tolerated following local intrastromal injection, with “virtually undetectable” off-target effects, supporting its potential for safe application in human subjects.

Clinical Trial Design and Objectives

The investigator-initiated trial is an open-label, dose-escalation study evaluating the tolerability of GEB-101 in combination with standard PTK treatment in adult patients diagnosed with TGFBI corneal dystrophy. The study aims to assess safety and inform the future clinical development pathway for the therapy.

Executive Statement on the Initiation of Clinical Study

Zongli Zheng, PhD, Chairman and Co-Founder of GenEditBio, expressed the significance of this clinical milestone:

“We are proud and honored to announce that we have initiated the world’s first clinical study of an in vivo genome editing investigational therapy for corneal dystrophy. This key milestone represents years of effort in technology platform development and dedication from our world-class research team and clinical partners. We stand at the frontier of a new era and recognize 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.”