GenEditBio Limited has achieved a significant milestone in genome editing therapeutics by dosing the first patient in an investigator-initiated trial of GEB-101, the world's first in vivo CRISPR-Cas ribonucleoprotein-based genome editing investigational therapy for TGFBI corneal dystrophy. The clinical-stage biotechnology company announced this breakthrough development in collaboration with Prof Xingtao Zhou and his team at the Eye & ENT Hospital of Fudan University in Shanghai, China.
The first patient received GEB-101 in May 2025 and was subsequently discharged from the collaborating hospital with no observable adverse events, marking a promising start for this pioneering therapeutic approach.
Novel CRISPR-Based Approach for Rare Eye Disease
TGFBI corneal dystrophy represents a group of genetic eye disorders caused by mutations in the TGFBI gene, resulting in abnormal protein buildup in the stromal layer of the cornea. Patients experience debilitating symptoms including photophobia, gradual vision loss, and recurrent corneal erosions. Current treatment options are limited to phototherapeutic keratectomy and corneal transplantation, both of which carry significant limitations including recurrence and risks of sight-threatening complications.
GEB-101 is designed as a once-and-done treatment that uses CRISPR-Cas genome editing technology to target specific loci in the mutated TGFBI gene. The therapy is encapsulated in ribonucleoprotein form within GenEditBio's proprietary engineered protein delivery vehicle (PDV), administered through intrastromal injection directly into the cornea.
Strong Preclinical Safety Profile
Preclinical assessment in non-human primates demonstrated that GEB-101 was well-tolerated after local intrastromal injection and exhibited a high safety profile with virtually undetectable off-target effects. This preclinical research recently received the Excellence in Research Award at the 28th Annual Meeting of the American Society of Gene and Cell Therapy in May 2025.
The ongoing investigator-initiated trial is structured as an open-label, dose-escalation clinical study investigating GEB-101's tolerability when combined with standard treatment phototherapeutic keratectomy in adults with corneal dystrophy.
Leadership Perspectives on Breakthrough Achievement
"Today, on National Sight Day, 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," said Zongli Zheng, PhD, Chairman and Co-Founder of GenEditBio. "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."
Tian Zhu, PhD, CEO and Co-Founder of GenEditBio, emphasized the rapid translation from laboratory to clinic: "Remarkably, in just one year since initiating our proof-of-concept and preclinical studies, we have successfully integrated our genome editing and delivery technologies to advance this pipeline program from the laboratory to clinical stage, showcasing our competence and dedication to rapidly translate basic research into clinical program to ultimately benefit patients."
Company Background and Technology Platform
Established in 2021 and headquartered in Hong Kong, GenEditBio focuses on developing potentially curative, once-and-done programmable in vivo genome editing-based therapeutic solutions, which the company refers to as "DNA surgery." The company's core areas of focus include novel Cas nuclease discovery and safe and efficient cargo delivery utilizing both lipid nanoparticle (LNP) and engineered protein delivery vehicle (PDV) technologies.
GenEditBio operates research laboratories and supporting offices in Hong Kong, Beijing, and Boston, with financial backing from top-tier life science investors including Qiming Venture Partners, Fangyuan Capital, Center Biotherapeutics, Lumosa Therapeutics, and HKSTP Venture Fund.
