GenEditBio gets FDA clearance for in vivo genome editing programme

HONG KONG, Jan. 5, 2026 /PRNewswire/ -- GenEditBio Limited ("GenEditBio"), a clinical-stage biotechnology startup focusing on genome-editing therapeutic solutions, today announced that the U.S. Food and Drug Administration (FDA) has cleared the company's Investigational New Drug (IND) application to initiate Phase 1/2 CLARITY trial activities for its lead in vivo genome-editing program GEB-101 for TGFBI corneal dystrophy.

The Phase 1/2 CLARITY trial will collect initial data on the safety, tolerability and efficacy of GEB-101 in corneal dystrophy patients with TGFBI mutation. The study has a seamless, adaptive, multicenter, sequential design. Trial participants will receive a single intrastromal injection of GEB-101. Patient enrollment is expected to commence in the second quarter of this year after site activation in the U.S.

"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 with high target tissue editing and low off-target editing risk to patients globally. This achievement reflects the concerted efforts of the entire company to advance preclinical assets into clinical stage with rigorousness, professionalism, and speed," said Zongli ZHENG, PhD, Chairman and Co-Founder of GenEditBio.

"GEB-101 is a first-in-class investigational genome-editing therapy for TGFBI corneal dystrophy. Current treatment options are limited and do not address the underlying cause, highlighting a significant unmet need for a targeted genetic approach. 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," added Tian ZHU, PhD, CEO and Co-Founder of GenEditBio.