GenEditBio Secures FDA Clearance for GEB-101 to Treat Corneal Dystrophy

GenEditBio Limited has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application, allowing the company to commence activities related to the Phase 1/2 CLARITY trial. This trial will evaluate the safety, tolerability, and efficacy of the company’s lead in vivo genome-editing program, GEB-101, specifically targeting TGFBI corneal dystrophy.

The CLARITY trial is designed as a multicenter, adaptive study that will include a seamless, sequential approach. Participants diagnosed with TGFBI mutation-related corneal dystrophy will receive a single intrastromal injection of GEB-101. Enrollment for the trial is anticipated 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 FDA’s approval, 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 emphasized the company’s dedication to advancing its preclinical assets into the clinical stage with rigor and speed.

Tian Zhu, PhD, CEO and Co-Founder, highlighted the significance of GEB-101 as a first-in-class investigational therapy for TGFBI corneal dystrophy. Current treatment options are limited and do not address the underlying genetic cause, illustrating a critical unmet need for targeted genetic approaches. “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,” Zhu added.

Understanding TGFBI Corneal Dystrophy

TGFBI corneal dystrophy encompasses a series of genetic eye disorders attributed to mutations in the TGFBI gene. These mutations lead to the accumulation of abnormal proteins in the cornea, resulting in symptoms such as photophobia, gradual vision loss, and recurrent corneal erosions. Patients often experience debilitating pain, severely impacting their quality of life.

Current treatment options, including phototherapeutic keratectomy (PTK) and corneal transplantation, present significant limitations, such as high recurrence rates and the risk of serious complications. The introduction of GEB-101 aims to fill this gap by providing a targeted genetic therapy that addresses the root cause of the condition.

About GEB-101 and GenEditBio

GEB-101 is a genome-editing drug candidate that employs CRISPR-Cas technology to target specific mutations in the TGFBI gene. This innovative treatment is encapsulated in ribonucleoprotein format within a proprietary protein delivery vehicle developed by GenEditBio, allowing for efficient in vivo delivery. The treatment is designed as a one-time administration, which could offer a significant advantage over current therapies.

Founded in 2021 and headquartered in Hong Kong, GenEditBio is dedicated to developing safe and effective genome-editing therapeutic solutions for genetic diseases. The company focuses on innovative Cas nuclease discovery and efficient cargo delivery systems, utilizing lipid nanoparticles and engineered protein delivery vehicles.

GenEditBio has garnered financial support from leading life science investors, including Qiming Venture Partners and Fangyuan Capital, among others. The company’s research facilities are located in Hong Kong, Beijing, and Boston, positioning it to make significant advancements in the field of gene therapy.

For further details, interested parties can visit the company’s website at www.geneditbio.com.