GenEditBio Secures FDA Clearance for Groundbreaking Gene Therapy

GenEditBio Limited has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application, enabling the company to initiate Phase 1/2 clinical trials for its innovative genome-editing program, GEB-101. This advancement specifically targets TGFBI corneal dystrophy, a genetic eye disorder that currently lacks effective treatment options.

The Phase 1/2 CLARITY trial aims to assess the safety, tolerability, and efficacy of GEB-101 in patients diagnosed with TGFBI mutations. This multicenter study employs a seamless, adaptive design, allowing for a more efficient evaluation of treatment outcomes. Participants will receive a single intrastromal injection of GEB-101, with patient enrollment expected to begin in the second quarter of 2026 following the activation of trial sites in the United States.

Zongli Zheng, PhD, Chairman and Co-Founder of GenEditBio, expressed enthusiasm regarding the IND clearance. He stated, “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.” Zheng emphasized the company’s dedication to advancing its preclinical assets into clinical stages with rigorous professionalism and speed.

Tian Zhu, PhD, CEO and Co-Founder of GenEditBio, highlighted the significance of GEB-101 as a first-in-class investigational therapy for TGFBI corneal dystrophy, noting that existing treatments do not address the underlying genetic cause of the disease. “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 is characterized by genetic mutations in the TGFBI gene, leading to progressive protein accumulation in the cornea. Patients often experience symptoms such as photophobia, gradual vision loss, and recurrent corneal erosions, which can significantly impact their quality of life. Current treatment modalities, including phototherapeutic keratectomy (PTK) and corneal transplantation, have notable limitations, such as high recurrence rates and potential complications that threaten vision. This underscores the urgent need for novel therapies that more effectively address the condition at its source.

About GEB-101 and GenEditBio

GEB-101 represents a pioneering approach as a genome-editing drug candidate designed for a one-time treatment of TGFBI corneal dystrophy. Utilizing CRISPR-Cas technology, GEB-101 targets a specific locus within the mutated TGFBI gene. The drug is encapsulated in ribonucleoprotein form, delivered via an engineered protein delivery vehicle (PDV), which is a proprietary system developed by GenEditBio.

Founded in 2021 and based in Hong Kong, GenEditBio focuses on providing in vivo genome-editing solutions, referred to as “DNA surgery.” The company aims to develop therapies that are safe, precise, and affordable for various genetic disorders. With research facilities in Hong Kong, Beijing, and Boston, GenEditBio is backed by prominent life science investors, including Qiming Venture Partners and Lumosa Therapeutics.

For further information, please visit the company’s website at www.geneditbio.com.