GenEditBio Secures FDA Approval for Groundbreaking Eye Treatment

GenEditBio Limited 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 proceed with the Phase 1/2 CLARITY trial for its lead in vivo genome-editing program, GEB-101, aimed at treating TGFBI corneal dystrophy. This disorder is characterized by genetic mutations that lead to progressive vision impairment due to abnormal protein deposits in the cornea.

The Phase 1/2 CLARITY trial is designed to assess the safety, tolerability, and preliminary efficacy of GEB-101 in patients with TGFBI mutations. The study features a seamless, adaptive, multicenter, and sequential design. Participants will receive a single intrastromal injection of GEB-101, with patient enrollment anticipated to begin in the second quarter of 2026 following site activation in the United States.

“This regulatory IND clearance for our lead clinical asset, GEB-101, marks a momentous milestone in our commitment towards bringing transformative ribonucleoprotein (RNP)-based therapy to patients globally,” stated Zongli Zheng, PhD, Chairman and Co-Founder of GenEditBio. He emphasized the company’s dedication to advancing its preclinical assets into clinical stages with rigorousness and speed.

GEB-101 represents a first-in-class investigational genome-editing therapy specifically targeting TGFBI corneal dystrophy. Current treatment options, such as phototherapeutic keratectomy (PTK) and corneal transplantation, have significant limitations, including high recurrence rates and potential complications. This highlights the urgent need for innovative therapies that address the underlying causes of this genetic disorder.

The CEO and Co-Founder, Tian Zhu, PhD, expressed optimism regarding the IND clearance, stating it validates their robust preclinical data on safety and efficacy. He added, “We look forward to trial site activation and plan to expand the CLARITY trial through regulatory clearance in other major markets.”

Understanding TGFBI Corneal Dystrophy

TGFBI corneal dystrophy is a group of genetic eye disorders stemming from mutations in the TGFBI gene. These mutations lead to the buildup of abnormal proteins in the cornea, resulting in symptoms such as photophobia, gradual vision loss, and recurrent corneal erosions that can cause debilitating pain. The impact on patients’ quality of life underscores the critical need for effective treatments.

Current management options are limited, often requiring multiple interventions. For instance, PTK involves removing the damaged corneal epithelium, while corneal transplantation is a more invasive procedure with extensive recovery times. Both approaches do not address the genetic root of the condition, emphasizing the necessity for a targeted genetic therapy such as GEB-101.

About GEB-101 and GenEditBio

GEB-101 is a pioneering genome-editing candidate developed by GenEditBio, utilizing CRISPR-Cas technology to specifically target the mutated TGFBI gene. Encapsulated as ribonucleoprotein using an engineered protein delivery vehicle (PDV), GEB-101 aims to provide a one-time treatment solution for TGFBI corneal dystrophy.

Founded in 2021 and headquartered in Hong Kong, GenEditBio is focused on developing in vivo genome-editing therapeutic solutions, often referred to as “DNA surgery.” The company aims to deliver safe, precise, and effective treatments for genetic diseases with unmet medical needs. It has research facilities in Hong Kong, Beijing, and Boston, and is supported by prominent life science investors, including Qiming Venture Partners and Fangyuan Capital.

For additional information, visit GenEditBio’s official website at www.geneditbio.com. For media inquiries, contact [email protected], and for investor relations, reach out to [email protected].