FDA Clears IND application for GenEditBio’s GEB-101 in TGFBI corneal dystrophy

The US Food and Drug Administration (FDA) has cleared GenEditBio Limited’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.¹

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 the cornea. Current treatment options include phototherapeutic keratectomy and corneal transplantation.^1,2

According to the company,¹ the CLARITY trial will collect initial data on the safety, tolerability, and efficacy of GEB-101 in corneal dystrophy patients with TGFBI mutation and is described as having a “seamless, adaptive, multicenter, sequential design.” Patients in the trial will receive a single intrastromal injection of GEB-101, and patient enrollment is expected to begin in Q2 of 2026.

GenEditBio describes GEB-101 as a first-in-class genome-editing drug candidate designed as a one-and-done treatment for TGFBI corneal dystrophy, based on the CRISPR-Cas genome-editing technology that targets a particular locus in the mutated TGFBI gene. GEB-101 is encapsulated as ribonucleoprotein in an engineered protein delivery vehicle (PDV).

Tian ZHU, PhD, CEO and co-founder of GenEditBio, commented on GEB-101, saying,¹ "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.”

In 2025, GenEditBio announced the first patient had been dosed in its investigator-initiated trial (IIT) of GEB-101. The IIT of GEB-101 is an open-label, dose-escalation clinical study to investigate the tolerability of GEB-101 when combined with standard treatment phototherapeutic keratectomy in adults with corneal dystrophy.

References:

1. GenEditBio receives FDA clearance of IND application for its lead in vivo genome-editing program GEB-101 for TGFBI corneal dystrophy. Published January 5, 2026. Accessed January 5, 2026. https://www.prnewswire.com/ae/news-releases/geneditbio-receives-fda-clearance-of-ind-application-for-its-lead-in-vivo-genome-editing-program-geb-101-for-tgfbi-corneal-dystrophy-302652376.html

2. Harp MD. GenEditBio doses first patient in investigator-initiated trial of GEB-101 for TGFBI corneal dystrophy. Published June 9, 2025. Accessed January 5, 2026. https://www.ophthalmologytimes.com/view/geneditbio-doses-first-patient-in-investigator-initiated-trial-of-geb-101-for-tgfbi-corneal-dystrophy