https://en.people.cn/n3/2025/0807/c90000-20350324.html

A team at the Beijing-based Chinese Institute for Brain Research has successfully treated a 61 years old female patient with retinitis pigmentosa (RP) through gene therapy. In 2022, the team discovered that opsin 5 (cOpn5), a unique photosensitive protein in chickens, exhibited exceptional sensitivity to visible light, especially within the blue-green spectrum. They developed an injectable solution using a recombinant adeno-associated virus (AAV) as a vector to carry the gene encoding this light-sensitive protein.

A single injection into the eye’s vitreous cavity of the patient triggered the expression of this protein in retinal ganglion cells, restoring their light sensitivity and partially returning vision to patients. Preclinical studies revealed that ganglion cells expressing this novel photosensitive protein could be effectively activated by natural light.  said Weng Danwei, R&D director at GenAns Biotech, the company that transformed Luo’s research into the gene therapy drug GA001.

“This significantly reduces the need for assistive devices and lowers phototoxicity risks, providing a safer, more convenient treatment that helps patients reconnect with the world,” Weng said.

In collaboration with GenAns Biotech, the company that transformed this research into the gene therapy drug GA001, Beijing Tiantan Hospital recruited 15 volunteer patients. The ability to perceive light significantly improved in the patients within a month, allowing them to distinguish larger objects like door frames and windows. By the second month, they could see smaller objects within a meter, such as fruits. Between three to six months, they had regained enough vision to live independently.

RP is a debilitating and hereditary condition that causes the gradual deterioration of retinal photoreceptor cells, leading to vision loss and, ultimately, blindness. The condition affects approximately 1 in 4,000 people, indicating a significant market demand for GA001.

Scientists have long sought ways to restore vision. In 2017, the U.S. approved Luxturna, the world’s first gene therapy for inherited retinal disease, marking a major milestone in ophthalmic treatment. However, Luxturna only targets one of the over 80 known gene mutations that cause RP, covering a mere 1  to 6 percent of RP patients, and only those in the early stages. In contrast, GA001 stands out as a broad-spectrum therapy that is effective across various mutations and even for patients in late stages of the disease, she added.

Currently, GenAns Biotech is conducting investigator-initiated trials for several gene therapies, including GA001 for blindness, GA002 for refractory epilepsy, and GA008 for intractable pain, aiming to revolutionize the treatment of refractory central nervous system disorders and retinal diseases worldwide.