At 61, a retinitis pigmentosa patient surnamed Zhao has regained her independence after losing her vision, thanks to a groundbreaking gene therapy injection that, within months, restored her ability to cook, clean and take walks with ease.

Before the treatment, the RP patient had severely limited sight due to her minimal perception of light and had to rely mainly on touch to navigate her surroundings.

But a breakthrough by a team led by Luo Minmin, a professor at the Beijing-based Chinese Institute for Brain Research, has changed Zhao's life.

In 2022, the team serendipitously discovered the remarkable potential of opsin 5, or cOpn5, a unique photosensitive protein derived from chickens. The protein exhibited exceptional sensitivity to visible light, especially within the blue-green spectrum.

Capitalizing on this discovery, Luo's team developed an injectable solution using a recombinant adeno-associated virus as a vector to carry the gene encoding the light-sensitive protein.

A single injection into the eye's vitreous cavity triggers the expression of the 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, research and development 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, Beijing Tiantan Hospital recruited 15 volunteer patients, including Zhao, who joined the trial in May 2024.

The treatment is simple and straightforward. Doctors use a fine needle to inject the drug into the eye's vitreous cavity. The entire process takes less than half an hour, requiring no invasive surgery or implanted devices.

According to Zhao, her ability to perceive light significantly improved within a month, allowing her to distinguish larger objects such as door frames and windows. By the second month, she could see smaller objects such as fruit within a meter. Between three to six months, she 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.

Weng noted that the condition affects approximately 1 in 4,000 people, indicating a significant market demand for GA001.

According to the China Association of Persons with Visual Disabilities, over 17 million people in China are visually impaired. As the population ages, the need for elderly care among blind individuals is becoming increasingly urgent.

At the 2025 Zhongguancun Forum Annual Conference, GA001 was recognized as one of the 10 major scientific and technological achievements in 2024 and is expected to progress to Phase I/II clinical trials by the end of this year.

Scientists have long sought ways to restore vision. In 2017, Luxturna, the world's first gene therapy for inherited retinal disease, marked a major milestone in ophthalmic treatment. Its use has been approved in the United States.

However, Weng said that Luxturna only targets one of the over 80 known gene mutations that cause RP, covering a mere 1 percent 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.

Xinhua