Der Pionier der Optogenetik, PAN Zhuohua, hat 125 Millionen US-Dollar eingesammelt.
Recently, the US biotechnology company in the clinical phase, Ray Therapeutics (hereinafter referred to as "Ray"), successfully completed a Series B financing of $125 million with oversubscription. This financing was led by the established asset manager Janus Henderson Investors, and Adage Capital Management, Franklin Templeton, Invus, Marshall Wace participated in the investment. Old shareholders such as 4BIO Capital, Deerfield Management, Merck's MRL Ventures Fund, Norwest, Novo Holdings A/S, Platanus and others have all increased their investments and shown long - term support with real money.
Financing progress diagram
These funds will be used to advance the core pipeline RTx - 015 through the late - stage clinical process for retinitis pigmentosa and pave the way for the commercialization of the product. At the same time, the clinical research of RTx - 021 for macular diseases such as Stargardt's disease and geographic atrophy will be accelerated to explore more application scenarios of the technology.
Nowadays, the number of participants in the field of ophthalmic gene therapy is constantly increasing, and different technology routes have their own focuses. In this competitive situation, why can Ray still gain the favor of investors?
Under the leadership of Zhuohua Pan, the technology problem is solved through the iteration of light - sensitive proteins
Degenerative retinal diseases are common causes of blindness worldwide. Retinitis pigmentosa and various macular diseases belong to this category. The causes of these diseases are complex. There are thousands of gene mutations just in retinitis pigmentosa. Therefore, traditional gene therapy is in a difficult situation: a drug can often only be developed for a single mutation, and the number of treatable patients is very limited. Currently, most common treatment methods can only slow down the degeneration of retinal cells, but cannot restore the lost vision. Once the disease enters the late stage, treatment is essentially impossible. At the same time, some advanced solutions require the use of special optical devices, which further increases the daily burden on patients.
In the face of these clinical challenges, the scientific community has long been seeking solutions. The Chinese - American scientist and pioneer in the field of visual optogenetics, Zhuohua Pan, is a forerunner in applying optogenetics technology to restore vision.
As early as the beginning of this century, Zhuohua Pan developed the idea: to modify the living neurons of the retina with light - sensitive proteins to replace the damaged photoreceptor cells and rebuild the visual signal pathway. In 2006, his team conducted in - vivo experiments on rodents for the first time and confirmed the feasibility of restoring vision through optogenetics worldwide for the first time. Later, he licensed the first - generation optogenetics patent to RetroSense Therapeutics, which conducted the world's first human clinical study in ophthalmic optogenetics based on this technology.
However, the light - sensitive protein ChR2 (Channelrhodopsin 2) used in the first generation has obvious weaknesses, including insufficient light sensitivity, activation only by strong light, and the potential risk of phototoxicity. Therefore, it is difficult to popularize this method on a large scale. After RetroSense was acquired by the global pharmaceutical company Allergan in 2016, the progress of this project gradually slowed down due to technological bottlenecks.
The step of scientific research did not stop there. In 2019, Zhuohua Pan's team published a key study in the journal "Molecular Therapy" and announced that it had achieved another technological breakthrough: The team carried out site - specific mutation optimization for the CoChR protein (a member of the Channelrhodopsin family) from Chloromonas oogama and selected two highly sensitive variants.
Figure F: Comparison curve of the vision restoration threshold under natural white light (Zhuohua Pan's team. Molecular Therapy, 2019, Figure 6.)
The experimental data show that the new - generation light - sensitive protein is free from the limitation of strong light and auxiliary means, can adapt to the daily light environment, and has excellent safety, which greatly compensates for the core weaknesses of the first - generation technology. Later, Ray, founded in 2021, licensed the improved technology patent and promoted its clinical implementation –
Ray's founder, Paul Bresge, has long specialized in the ophthalmic field. Due to the retinitis pigmentosa in his family, he has always been committed to finding solutions for the causes of blindness. The core members of the team have also witnessed the early development of optogenetics. The chief scientist Peter Francis led the clinical work on the first - generation therapy at RetroSense and has a deep understanding of technological iteration and clinical problems. This team, which has both scientific vision, clinical experience, and commercial capabilities, is a solid force for the implementation of the new technology.
Based on this optogenetic platform for the visual system, which has been refined in two rounds, Ray has built a differentiated treatment system. The team uses the minimally invasive intravitreal injection method and transports the gene encoding the improved CoChR light - sensitive protein into retinal neurons with the help of an adeno - associated virus vector. The cells that originally had no light sensitivity are newly modified and can perceive natural light and convert the light signal into an electrical signal that is sent to the brain, thus rebuilding the complete visual signal pathway.
Compared with traditional therapy, this method has obvious advantages: it is not dependent on the type of gene mutation, breaks the limitation of a single drug for a single mutation, and is suitable for a wider population. The treatment goal is not to slow down the disease process, but to directly restore the visual signal pathway. Even patients in the late stage with complete apoptosis of photoreceptor cells have a treatment option. The therapy only requires a single injection and promises long - term effects. No additional optical equipment or drugs are required, which greatly simplifies the treatment and care process.
Two pipelines cover different retinal diseases
Based on the mature optogenetics technology, Ray has planned two complementary pipelines to carry out clinical development for different lesion cells and eye diseases.
As the core pipeline, RTx - 015 targets retinal ganglion cells and is administered through a single intravitreal injection. The core indication, retinitis pigmentosa, has already reached the Phase II/III stage, and at the same time, a Phase I clinical study is being conducted for other hereditary retinal diseases such as congenital chorioretinitis. Due to the positive early clinical data, RTx - 015 received both the Regenerative Medicine Advanced Therapy (RMAT) qualification from the US FDA and the Priority Medicines (PRIME) qualification from the European EMA in 2026, which enables it to benefit from regulatory advice and priority review, and the research rhythm is continuously accelerated.
The RTx - 021 pipeline pursues a differentiated targeting strategy and targets retinal ON - bipolar cells. It focuses on macular diseases such as Stargardt's disease and geographic atrophy, which occurs secondary to age - related macular degeneration. These diseases mainly damage the macular region of the eye and directly affect a person's central vision. They are an important cause of blindness. The Phase I clinical study of RTx - 021 for Stargardt's disease has already started. The core goal is to validate its ability to repair central vision and fill the gap in the treatment of macular diseases.
Current pipelines (Image from Ray's official website)
The two pipelines are related to each other and have different targets. They can maximize the reuse value of the technology platform and reduce the research risk at the same time. On the production side, Ray has built a cGMP production system with Forge Biologics. Forge Biologics undertakes the cGMP production of AAV vectors and plasmid DNA for Ray, which can provide stable capacity support for the clinical further development of the pipelines and subsequent commercialization.
The ice and fire of retinal optogenetic therapy
Currently, ophthalmic optogenetic therapy is in a critical transition area from clinical research to commercialization. Many companies around the world have positioned themselves in this field, and different technology solutions and research progress have gradually heated up the market competition. This technology goes beyond the boundaries of traditional gene therapy and is not bound to specific gene mutations. It particularly meets the treatment needs of patients with advanced retinal diseases, and the value of the field's advantage is gradually becoming visible.
Incomplete statistics from Arterial Network on the pipelines of ophthalmic optogenetic therapy at home and abroad
Overall, in this field, Western countries started earlier and China is catching up quickly. Western companies have conducted research in this field for many years and have a strong technological foundation. Among them, Nanoscopes MCO - 010 has made the most progress and has the best chance of being the first to be brought to the market. The approval decision and clinical performance after market entry could become a guide for the entire field. The early pipelines of companies such as AbbVie, GenSight, Bionic Sight are limited due to the insufficient sensitivity of the first - generation light - sensitive proteins and require the use of optical goggles to be effective. Restore Vision has taken a differentiated GPCR - optogenetic route and developed a new generation of chimeric rhodopsin proteins, which may be freed from the dependence on external aids.
Chinese companies entered the field later, but they have a high technological starting point and strong catching - up momentum: The GA001 pipeline of Jianda Jiuzhou has already received the FDA's Fast Track qualification and Orphan Drug Designation and is one of the fastest - progressing pipelines in Chinese optogenetic gene therapy. Zhongmou Medicals' ZM - 02 has planned clinical studies in both China and the United States at the same time. The exploratory clinical study led by Peking Tongren Hospital has completed the 52 - week long - term follow - up and received the IND approval from the FDA in November 2025 to start the international multicenter clinical study. Xingming Youjian's UGX - 202 has completed the first administration in the exploratory clinical study. The application for Phase I registration was accepted by the CDE in May 2026, and at the same time, it licensed the overseas rights to the British company AviadoBio to start a global strategy.
However, although the field is getting hotter, as of June 2026, no ophthalmic optogenetic drug has been brought to the market worldwide, and the entire field is still on the eve of commercialization. Technological weaknesses, regulatory rules, production costs and other problems are also common challenges for all players.
Technologically, the immune risk of the AAV vector and the long - term safety of the exogenous light - sensitive protein still need to be validated through long - term clinical studies. The clinical efficacy evaluation system does not yet have a global unified standard. The regulatory rules for the cross - cutting fields of gene therapy and neuronal regulation are still being improved, which further prolongs the research and review period. At the same time, strict production standards increase production costs. Commercial issues such as future product pricing and market cultivation also need to be solved by the industry.
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