Rewriting the Code of Sight: A New Dawn for Corneal Dystrophy Treatment
- GenEditBio has dosed the first patient in a groundbreaking trial for GEB-101, a novel gene-editing therapy.
- This marks the world's pioneering clinical study of an in vivo CRISPR-Cas ribonucleoprotein (RNP)-based therapy for TGFBI corneal dystrophy.
- The therapy, GEB-101, aims to be a "once-and-done" treatment, potentially revolutionizing care for this genetic eye disorder.
A new era in genetic medicine has dawned. GenEditBio Limited, a trailblazing clinical-stage biotechnology company, today announced a monumental leap forward: the first patient has been treated with GEB-101, the world's first in vivo CRISPR-Cas RNP-based investigational therapy designed to combat TGFBI corneal dystrophy. This devastating group of genetic eye disorders, caused by mutations in the TGFBI gene, leads to abnormal protein buildup, photophobia, recurrent corneal erosions, and progressive vision loss2, 5. Current treatments like phototherapeutic keratectomy (PTK) and corneal transplants offer temporary relief but come with limitations, recurrence, and risks3, 4.
GEB-101, administered via a local intrastromal injection, is engineered as a "DNA surgery" to precisely target and edit the mutated gene, offering the potential for a permanent, curative solution. Preclinical studies in non-human primates showcased GEB-101's high safety profile with virtually undetectable off-target effects, research so promising it earned the Excellence in Research Award at the American Society of Gene and Cell Therapy's 28th Annual Meeting.
The ongoing investigator-initiated trial, in collaboration with Prof Xingtao ZHOU and his team at the Eye & ENT Hospital of Fudan University, is an open-label, dose-escalation study evaluating GEB-101 with standard PTK7. The first patient has already been discharged with no observable adverse events, a significant early sign of hope.
"Today, on National Sight Day, we are proud and honored," declared Zongli ZHENG, PhD, Chairman and Co-Founder of GenEditBio. "We stand at the frontier of a new era... the transformative potential of this moment is not just for the Company but for the entire field of genetic medicine."
Tian ZHU, PhD, CEO and Co-Founder, added, "Remarkably, in just one year... we have successfully integrated our genome editing and delivery technologies to advance this pipeline program from the laboratory to clinical stage... As clinical data accumulate, we are positive that GEB-101 has the potential to become a new treatment option."
This pivotal trial signifies more than just a corporate milestone; it represents a beacon of hope for patients worldwide, heralding a future where genetic vision loss might be rewritten at its very source6, 7. GenEditBio is committed to making these advanced therapies safe, efficacious, and accessible.
For more information, please visit www.geneditbio.com.
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