GenEditBio Gains FDA Clearance for Groundbreaking Gene Therapy

GenEditBio Limited, a biotechnology startup based in Hong Kong, has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application. This approval allows the company to commence Phase 1/2 trials of its lead in vivo genome-editing program, known as GEB-101, aimed at treating TGFBI corneal dystrophy. The announcement was made on January 5, 2026.

The upcoming Phase 1/2 CLARITY trial will evaluate the safety, tolerability, and preliminary efficacy of GEB-101 in patients diagnosed with TGFBI mutation-related corneal dystrophy. This multicenter trial features an adaptive design, with participants receiving a single intrastromal injection of GEB-101. Enrollment is anticipated to begin in the second quarter of 2026 following the activation of trial sites across the United States.

Zongli Zheng, PhD, Chairman and Co-Founder of GenEditBio, emphasized the significance of this regulatory milestone, 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, ready-to-act and rapid degradation editor for one-and-done in vivo genome-editing therapy.” Zheng highlighted the company’s dedication to advancing its preclinical assets into clinical stages with professionalism and speed.

Tian Zhu, PhD, CEO and Co-Founder of GenEditBio, added that GEB-101 represents a first-in-class investigational therapy for TGFBI corneal dystrophy. Currently available treatment options are limited and fail to address the root cause of the disease, creating a significant unmet need for targeted genetic therapies. Zhu noted, “This IND clearance validates our robust preclinical data on safety and efficacy.”

Understanding TGFBI Corneal Dystrophy

TGFBI corneal dystrophy is characterized by genetic mutations in the TGFBI gene, leading to abnormal protein accumulation in the cornea. Patients often experience debilitating symptoms, including sensitivity to light, gradual vision loss, and painful recurrent corneal erosions. Existing treatments, such as phototherapeutic keratectomy and corneal transplantation, present limitations, including high recurrence rates and potential complications that threaten vision.

About GEB-101 and GenEditBio

GEB-101 is a pioneering genome-editing candidate designed as a one-time treatment for TGFBI corneal dystrophy. Utilizing CRISPR-Cas technology, GEB-101 targets a specific location in the mutated TGFBI gene. The treatment is encapsulated in ribonucleoprotein form, delivered via an engineered protein delivery vehicle developed by GenEditBio.

Founded in 2021, GenEditBio aims to provide innovative in vivo genome-editing solutions, referred to as “DNA surgery,” for genetic diseases with unmet clinical needs. The company operates research laboratories in Hong Kong, Beijing, and Boston, and is supported by prominent life science investors, including Qiming Venture Partners and Fangyuan Capital.

For further information about GenEditBio, visit their official website at www.geneditbio.com.