[China Tech] FDA Clears Gene-Editing Trial for Corneal Dystrophy at Fudan University Hospital

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A local hospital's clinical trial for a gene-editing therapy to treat corneal dystrophy has received Investigational New Drug (IND) approval from the US Food and Drug Administration.

Corneal dystrophy is a blinding hereditary eye disease and can be challenging because traditional treatments cannot prevent recurrence.

The research led by Fudan University's Eye, Ear, Nose and Throat Hospital professors Hong Jiaxu and Zhou Xingtao developed a novel drug for hereditary corneal dystrophy gene-editing therapy, GEB-101, which is currently under a clinical study at the hospital.

This drug is the first in vivo genome-editing treatment for TGFBI-related corneal dystrophy. After approval, the R&D parties will launch "CLARITY" in the US and China as a Phase I/II clinical trial. The hospital said an international regulatory agency recognized a Chinese-developed ophthalmic gene therapy drug.

TGFBI gene mutations cause autosomal dominant hereditary corneal dystrophy, which causes abnormal protein deposition in the cornea. Patients develop severe photophobia, recurrent eye pain and progressive vision loss, eventually blinding them.

Traditional treatments like phototherapeutic keratectomy (PTK) or corneal transplantation can only temporarily remove deposits or replace the cornea but cannot prevent disease recurrence at the genetic level, burdening patients with the need for multiple surgeries.

GEB-101, as an innovative in vivo genome-editing therapy, aims to target the pathogenic gene at its root, achieving efficient and precise editing while pursuing a lower risk of off-target effects. It holds the promise of offering patients an entirely new, potentially curative treatment option.

The hospital expects the newly approved trial to begin patient enrollment at US research centers in the second quarter of this year, followed by Chinese studies.

If you want to consult and participate in the clinical trial

You can visit ophthalmology department of the hospital in both Xuhui and Pujiang branches.

Xuhui branch: 83 Fenyang Rd (汾阳路83号)

Pujiang branch: 2600 Jiangyue Rd (江月路2600号)