The Nobel Prize in Physiology or Medicine brings optogenetics into the spotlight.
On October 5, local time, the Swedish Karolinska Institute announced that the 2026 Nobel Prize in Physiology or Medicine would be awarded to American scientist Carl Deisseroth and German scientists Peter Hagermann and Georg Nagel in recognition of their discoveries in light-gated ion channels and optogenetics.
According to the Nobel Prize official website, Hegerman and Nagel discovered a special protein called channelrhodopsin in a single-cell algae. When this protein on the cell surface is illuminated by blue light, ion channels inside it open like gates, allowing charged ions to enter the cell and generate electrical signals. They also found that placing the protein in other cells could make those cells respond to light. Deisseroth then introduced the gene encoding channelrhodopsin into rat nerve cells and successfully triggered nerve signals through blue light irradiation. He published this research result in 2005, and two years later he further achieved the use of light to control nerve cell activity in the brains of living mice.
This method, known as optogenetics, allows researchers to study how nerve cells in living brains influence memory, sensation and behavior, and to reveal neural circuits associated with neurological and psychiatric disorders.
Pan Qiang, a judge of the Nobel Prize in Physiology or Medicine and a professor of clinical immunology at the Karolinska Institute in Sweden, said in an interview with a reporter from Xinhua News Agency that optogenetics provides an important tool for studying brain function and also shows potential for clinical application. Researchers have tried to use this technology to help patients with certain retinal diseases restore some visual function and to study the neural mechanisms behind diseases such as depression, Alzheimer's disease and pain.
Optogenetics has also become one of the technical paths for research and development by domestic and foreign pharmaceutical companies. Its coverage areas are mainly in the two fields of ophthalmology and central nervous system diseases, among which blinding eye diseases such as retinitis pigmentosa (RP) are the focus of competition.
In September this year, the U.S. FDA accepted the Biologics License Application (BLA) for its optogenetic gene therapy MOGENRY, which is intended to be used to treat RP patients with severe vision loss. MOGENRY is a one-time intravitreal injection of optogenetic gene therapy. If successfully approved, it is expected to be the first therapy for this patient group that does not need to target a specific disease-causing gene.
Back in 2016, Allergan, which was later acquired by AbbVie in 2019, announced the acquisition of RetroSense Therapeutics for a down payment of US$60 million. This company is developing a gene therapy using optogenetic technology to treat patients who are blind due to retinitis pigmentosa.
In 2021, Novartis acquired a preclinical optogenetic AAV gene therapy program and Arctos' proprietary technology through the acquisition of Arctos Medical. Public information at the time showed that Arctos used adeno-associated virus (AAV) gene therapy technology to deliver light genes to specific retinal cells and express them, thereby replacing the function of photoreceptor cells. It mainly developed potential methods to treat inherited retinal dystrophy (IRD) and other diseases involving photoreceptor loss (such as AMD).
Domestically, a number of start-up companies are also quietly increasing their investment, including Xingming Youjian, Jianda Jiuzhou, Zhongmu Medical, etc., backed by listed pharmaceutical companies such as Fosun Pharma (600196).
In October 2025, UgeneX and AviadoBio reached an exclusive option and license agreement for the development and commercialization of the optogenetic pipeline UGX-202. Under the agreement, AviadoBio has the option to obtain an exclusive global license to develop and commercialize UGX-202 for RP (retinitis pigmentosa) and other indications outside Greater China. If the option is exercised, UgeneX will be eligible to receive up to $413 million in upfront payments, R&D milestone payments and sales milestone payments, as well as royalties on net sales.
Public information shows that UGX-202 is an adeno-associated virus (AAV) gene therapy in the clinical development stage for the treatment of RP patients. When the above-mentioned transaction was officially announced, Fosun Pharma also issued a statement stating that Xingming Youjian is a biotechnology company focusing on innovative gene therapies for ophthalmic diseases incubated in July 2021 by the "Rehabilitation Capital New Drug Innovation Fund" initiated and established by Fosun Pharma. Tianyancha shows that Suzhou Fuxing Xingyi Venture Capital Partnership (Limited Partnership) holds 50.8222% of the company's equity, and Tianjin Fosun Haihe Medical and Health Industry Fund Partnership (Limited Partnership) holds 25.3996% of the company's equity.
In July this year, Kinder Jiuzhou announced the completion of hundreds of millions of yuan in Series A financing. The company's existing pipeline has deeply covered major indications such as retinopathy, epilepsy, and central neuralgia, and the backup pipeline is further extending into high-barrier areas with unmet needs such as Parkinson's disease, severe depression, drug addiction, and malignant brain tumors. The core pipeline of optogenetics, GA001, targets retinitis pigmentosa and has received Phase II clinical approval and orphan drug qualification and fast track qualification from the US FDA. Registration clinical trials were officially launched in July.
Tianyancha information shows that Jianda Jiuzhou’s shareholders include Beinao Phase I (Beijing) Equity Investment Center (Limited Partnership), and the center’s partners include A-share listed companies such as Zhaoyan New Drug (603127) and Sanbo Brain Science (301293). Participants in the above-mentioned financing include Biotech (688767), which participated in this round of investment through the Biotech Brain-Computer Fund established by it.
ZM-02, ZM Medical’s core product, a new generation of optogenetic gene therapy, is used to treat advanced retinal degenerative diseases. It was approved for clinical trials by the State Food and Drug Administration in August this year. It was previously granted orphan drug status and clinical approval by the US FDA. The company is backed by the listed company Hitech Biotechnology (300683). Tianyancha shows that Wuhan Haite Bio-Innovative Pharmaceutical Research Co., Ltd., a wholly-owned subsidiary of Haite Biotechnology, holds 15.0769% of the shares of Zhongmu Medical.
With the blessing of the Nobel Prize, the track of optogenetics is expected to trigger a new craze in the pharmaceutical industry.
