UM research team maps global patent landscape of optogenetics to facilitate clinical translation

MACAU, May 27 - A research team led by Professor Hu Yuanjia in the Institute of Chinese Medical Sciences (ICMS) and the State Key Laboratory of Mechanism and Quality of Chinese Medicine at the University of Macau (UM), in collaboration with Zhang Yanfei, investigator at the Tianjin Institute of Industrial Biotechnology of the Chinese Academy of Sciences, has made new progress in analysing the global patent landscape of optogenetics. The team has systematically mapped the technological evolution, milestone patents, industry competition, key challenges, clinical breakthroughs, and translational prospects in the field, providing insights that may inform the development of novel, clinically applicable therapeutic approaches. The research findings have been published in the academic journal Nature Biotechnology.

Optogenetics is a biotechnology that uses photosensitive proteins to enable precise regulation of cellular activities, allowing for the modulation of specific biological processes on a millisecond timescale and at the single-cell level. Initially used primarily in neuroscience research, optogenetics has gradually expanded in recent years to include synthetic biology, natural product biosynthesis, and clinical medicine. With advances in photosensitive protein engineering and gene delivery systems, optogenetics has entered multiple clinical trials, particularly showing strong translational potential in the treatment of retinal diseases. However, no optogenetics-related products have yet been approved for marketing, suggesting that critical bottlenecks remain in translating this technology from basic research into industrial and clinical applications. As an important bridge between technological innovation and industrial applications, patents can systematically reflect technological evolution, innovation strategies, and industrial competition, making them a key data resource for identifying translational barriers and guiding research and development.

Based on a systematic analysis of 332 patent families, the research team found that after a period of sustained growth, the number of optogenetics patent applications peaked in 2019, before experiencing a notable decline. Blue light-activated optogenetic tools accounted for 41% of all innovations in terms of technological composition, and have been widely applied in clinical medicine and cytobiology. As of the end of 2023, 11 clinical trials had been registered, with a sustained focus on the treatment of retinal diseases. This represents the most promising clinical breakthrough for optogenetics. However, the decline in patent applications suggests deeper obstacles in the process of industrialisation. At the technical level, existing optogenetic systems still face inherent limitations in tissue penetration, photosensitive protein performance, and long-term expression stability. Meanwhile, safety data for human applications remain insufficient, and adaptive regulatory frameworks across different technology categories have yet to be established, and ethical concerns are intensifying the challenges of clinical translation.

Breakthroughs in this field may no longer depend solely on the optimisation and iteration of individual technologies, but also on multidisciplinary integration. The combination of cutting-edge technologies such as the precise regulation of calcium channels, upconversion luminescence materials, and organoid systems can enable optogenetics to overcome existing bottlenecks and explore new therapeutic paradigms with clinical feasibility. In addition, the latest data indicates that, following a continuous decline throughout 2023, the number of optogenetics-related patent publications increased rapidly in 2024 and 2025. Notably, a few new patents have been published in the field of oncology, involving light-controlled immune cell infiltration into solid tumours and near-infrared light-regulated STING-related innate immune pathways, suggesting potential applications in tumour immune regulation. According to the latest statistics, the number of related clinical trials has further increased to 16. Among them, MCO-010 developed by Nanoscope Therapeutics is leading in development, having completed a Phase IIb clinical trial and initiated a rolling submission of a Biologics License Application (BLA) to the US Food and Drug Administration (FDA).

The corresponding authors of this study are Prof Hu and Dr Zhang Yanfei, while the co-first authors are Zhang Yufan, a PhD student at UM, and Lyu Liyang, a PhD graduate of UM (currently an associate professor in the School of International Pharmaceutical Business at China Pharmaceutical University). The research was funded by the Science and Technology Development Fund of the Macao SAR (Grant Nos.: 0002/2025/NRP, 0049/2024/AGJ), the University of Macau (Grant Nos.: MYRG-CRG2023-00007-ICMS-IAS, MYRG-GRG2024-00268-ICMS), the University of Macau–Dr. Stanley Ho Medical Development Foundation AI+ Applied Research Grant (Grant No.: SHMDF-AI/2026/003), the Tianjin Synthetic Biotechnology Innovation Capacity Improvement Project (Grant No.: TSBICIP-IJCP-004), and the Tianjin Major Science and Technology Projects and Engineering Programs (National Key Laboratory Projects) (Grant No.: 25ZXZSSS00010). Patent data support was provided by Derwent Innovation (DI) of the UM Library. The full version of the research article can be accessed at: https://doi.org/10.1038/s41587-026-03080-w.