GenEditBio Receives FDA Clearance of IND Application for Its Lead In Vivo Genome-Editing Program GEB-101 for TGFBI Corneal Dystrophy

HONG KONG,Jan. 5, 2026/PRNewswire/ -- GenEditBio Limited ("GenEditBio"), a clinical-stage biotechnology startup focusing on genome-editing therapeutic solutions, today announced that the U.S. Food and Drug Administration (FDA) has cleared the company's Investigational New Drug (IND) application to initiate Phase 1/2 CLARITY trial activities for its lead in vivo genome-editing program GEB-101 for TGFBI corneal dystrophy.

The Phase 1/2 CLARITY trial will collect initial data on the safety, tolerability and efficacy of GEB-101 in corneal dystrophy patients with TGFBI mutation. The study has a seamless, adaptive, multicenter, sequential design. Trial participants will receive a single intrastromal injection of GEB-101. Patient enrollment is expected to commence in the second quarter of this year after site activation in the U.S.

"This regulatory IND clearance for our lead clinical asset, GEB-101, marks a momentous milestone in our commitment towards bringing transformative ribonucleoprotein (RNP)-based, ready-to-act and rapid degradation editor for one-and-done in vivo genome-editing therapy with high target tissue editing and low off-target editing risk to patients globally. This achievement reflects the concerted efforts of the entire company to advance preclinical assets into clinical stage with rigorousness, professionalism, and speed,"said Zongli ZHENG, PhD, Chairman and Co-Founder of GenEditBio.

"GEB-101 is a first-in-class investigational genome-editing therapy for TGFBI corneal dystrophy. Current treatment options are limited and do not address the underlying cause, highlighting a significant unmet need for a targeted genetic approach. This IND clearance validates our robust preclinical data on safety and efficacy. We look forward to trial site activation and plan to expand the CLARITY trial through regulatory clearance in other major markets,"added Tian ZHU, PhD, CEO and Co-Founder of GenEditBio.

About TGFBI Corneal Dystrophy

TGFBI corneal dystrophy is a group of genetic eye disorders caused by mutations in theTGFBIgene, resulting in progressive abnormal protein buildup and deposits in the cornea. Symptoms include photophobia, gradual vision loss and recurrent corneal erosions with debilitating pain, impacting patients'long-term quality of life. Current treatment options include phototherapeutic keratectomy (PTK) and corneal transplantation. Both procedures, however, have known limitations such as high rates of recurrence and the risk of sight-threatening complications, underscoring the urgent need for novel therapies.

About GEB-101

GEB-101, a wholly owned program of GenEditBio, is a first-in-class genome-editing drug candidate designed as a one-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 ribonucleoprotein in engineered protein delivery vehicle (PDV), a proprietary in vivo delivery system developed by GenEditBio. GEB-101 is to be administered by a single intrastromal injection and is being investigated for safety and preliminary efficacy in an investigator-initiated trial (IIT) in China.

About GenEditBio

Established in 2021 and headquartered in Hong Kong, China, GenEditBio is a clinical-stage gene therapy startup with a strategic goal of providing in vivo genome-editing therapeutic solutions (dubbed"DNA surgery") that are fundamentally safe, precise, efficacious, and affordable 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 visitwww.geneditbio.com.

Media Contact

geb.media@geneditbio.com

Investors Contact

bd@geneditbio.com

View original content to download multimedia:https://www.prnewswire.com/apac/news-releases/geneditbio-receives-fda-clearance-of-ind-application-for-its-lead-in-vivo-genome-editing-program-geb-101-for-tgfbi-corneal-dystrophy-302652326.html

SOURCE GenEditBio Limited