GenEditBio Limited ("GenEditBio"), a clinical-stage biotechnology start-up company focusing on genome editing therapeutic solutions through the discovery of novel and precise Cas nucleases and the development of safe and efficient cargo delivery platforms, announced a key milestone in the development of its pipeline: First patient has been dosed in an investigator-initiated trial (IIT) of GEB-101, the Company's leading in vivo genome editing program for TGFBI corneal dystrophy. The ongoing IIT is in collaboration with Prof Xingtao ZHOU, MD, PhD, Professor and President, and his team at the Eye & ENT Hospital of Fudan University in Shanghai, China.

GEB-101 is a wholly owned program of GenEditBio. Preclinical assessment in non-human primates demonstrated that GEB-101 was well-tolerated after local intrastromal injection and had high safety profile with virtually undetectable off-target effect (https://www.geneditbio.com/newsinfo/8296786.html). The preclinical research recently received the Excellence in Research Award at the 28^th Annual Meeting of the American Society of Gene and Cell Therapy in May 2025 (https://www.geneditbio.com/newsinfo/8348801.html).

The IIT of GEB-101 is an open-label and dose-escalation clinical study to investigate the tolerability of GEB-101 when combined with standard treatment phototherapeutic keratectomy in adults with corneal dystrophy. The first patient, who received GEB-101 in May 2025, has been discharged from the collaborating hospital with no observable adverse event. This trial marks the world's first clinical study of an in vivo CRISPR-Cas ribonucleoprotein (RNP)-based genome editing investigational therapy for TGFBI corneal dystrophy.

"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. This key milestone represents years of effort in technology platform development and dedication from our world-class research team and clinical partners," 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. Our company is committed to developing fundamentally safe, efficacious and affordable in vivo genome editing therapies for genetic diseases with unmet medical needs".   

"This investigator-initiated trial marks a defining moment for our award-winning research team and clinical partners. 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," said Tian ZHU, PhD, CEO and Co-Founder of GenEditBio, "We also express our deepest gratitude to the participant who volunteers in this early stage trial to support medical innovation. As clinical data accumulate, we are positive that GEB-101 has the potential to become a new treatment option for patients with corneal dystrophy".