GenEditBio Announces First Patient Dosed in Investigator-Initiated Trial of GEB‑101, World's First In Vivo CRISPR-Cas Ribonucleoprotein-Based Genome Editing Investigational Therapy for TGFBI Corneal Dystrophy
HONG KONG, BEIJING and BOSTON, June 6, 2025 /PRNewswire/ -- 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, today 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 28th 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".
About TGFBI Corneal Dystrophy
TGFBI corneal dystrophy is a group of genetic eye disorders caused by mutations in the TGFBI gene, resulting in abnormal protein buildup in the stromal layer of cornea. Symptoms include photophobia, gradual vision loss and recurrent corneal erosions. Current treatment options include phototherapeutic keratectomy and corneal transplantation. These procedures, however, have known limitations such as recurrence and carry risks of sight-threatening complications, underscoring the need for novel therapies.
About GEB-101
GEB-101, a wholly owned program of GenEditBio, is a genome editing drug candidate designed as a once-and-done treatment for TGFBI corneal dystrophy. GEB-101 is based on the CRISPR-Cas genome editing technology that targets a particular locus in the mutated TGFBI gene. GEB-101 is encapsulated in the form of RNP in engineered protein delivery vehicle (PDV), a proprietary in vivo delivery system developed by GenEditBio. GEB-101 is to be administered by intrastromal injection and is being investigated for tolerability in the IIT.
About GenEditBio
Established in 2021 and headquartered in Hong Kong, China, GenEditBio is a gene therapy start-up company with an overarching strategic goal of providing potentially curative, once-and-done and programmable in vivo genome editing-based therapeutic solutions (dubbed "DNA surgery") with high safety profile, unmatched precision, and affordable access for genetic diseases with unmet needs. The Company's core areas of focus include novel Cas nuclease discovery and safe and efficient cargo delivery utilizing lipid nanoparticle (LNP) and engineered protein delivery vehicle (PDV). We have research laboratories and supporting offices in Hong Kong, Beijing, and Boston. GenEditBio is financially backed by top-tier life science investors, including Qiming Venture Partners, Fangyuan Capital, Center Biotherapeutics, Lumosa Therapeutics, HKSTP Venture Fund, and others. For more information, please visit www.geneditbio.com.
Related Previous Press Releases
- GenEditBio Scientist Receives Excellence in Research Award at the 28th Annual Meeting of the American Society of Gene and Cell Therapy 2025 https://www.geneditbio.com/newsinfo/8348801.html
- GenEditBio to Present Preclinical Data on its Delivery Technology Platform and Drug Pipeline at the 28th Annual Meeting of the American Society of Gene and Cell Therapy https://www.geneditbio.com/newsinfo/8296786.html
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