GenEditBio Secures FDA Approval for GEB-101 Trial in Corneal Dystrophy

GenEditBio Limited has achieved a significant milestone with the recent clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application. This approval allows the company to initiate Phase 1/2 clinical trial activities for its lead in vivo genome-editing program, GEB-101, aimed at treating TGFBI corneal dystrophy. The trial, known as CLARITY, will assess the safety, tolerability, and efficacy of GEB-101 in patients diagnosed with this genetic eye disorder.

The CLARITY trial is designed as a seamless, adaptive, multicenter study. Participants will receive a single intrastromal injection of GEB-101, with patient enrollment expected to begin in the second quarter of 2026 following site activation in the United States.

Zongli Zheng, PhD, Chairman and Co-Founder of GenEditBio, expressed enthusiasm about the IND clearance, stating, “This regulatory IND clearance for our lead clinical asset, GEB-101, marks a momentous milestone in our commitment towards bringing transformative ribonucleoprotein (RNP)-based therapies to patients globally.” He highlighted the company’s dedication to advancing their preclinical assets into the clinical stage with rigor and speed.

Dr. Tian Zhu, CEO and Co-Founder, further emphasized the significance of GEB-101, describing it as a first-in-class investigational therapy for TGFBI corneal dystrophy. Current treatment options are limited and fail to address the underlying genetic causes of the disorder, creating a critical need for targeted genetic therapies. “This IND clearance validates our robust preclinical data on safety and efficacy,” said Dr. Zhu. He looks forward to expanding the CLARITY trial into other major markets once regulatory clearances are obtained.

TGFBI Corneal Dystrophy Overview

TGFBI corneal dystrophy is a genetic condition caused by mutations in the TGFBI gene, leading to the progressive accumulation of abnormal proteins in the cornea. Patients typically experience symptoms such as photophobia, gradual vision loss, and recurrent corneal erosions, which can cause debilitating pain and significantly impact their quality of life.

Current treatment options include phototherapeutic keratectomy (PTK) and corneal transplantation. Both procedures carry risks of complications and recurrence, underscoring the urgent need for more effective therapies that target the root cause of the condition.

About GEB-101 and GenEditBio

GEB-101 is a groundbreaking genome-editing drug candidate developed by GenEditBio. It utilizes CRISPR-Cas technology to target specific mutations in the TGFBI gene. The therapy is delivered via a proprietary engineered protein delivery vehicle, designed to ensure precise and effective treatment through a single injection.

Founded in 2021 and based in Hong Kong, GenEditBio is dedicated to providing innovative in vivo genome-editing therapeutic solutions. The company focuses on addressing genetic diseases with significant unmet needs, employing cutting-edge technologies to enhance safety and efficacy.

GenEditBio is supported by prominent investors in the life sciences sector, including Qiming Venture Partners, Fangyuan Capital, and others. For more information, visit their website at www.geneditbio.com.

As the company prepares for the upcoming trial, it anticipates that GEB-101 could represent a transformative approach in the treatment of corneal dystrophy, offering hope to patients in need of effective therapies.