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Outline
Rare diseases are typically characterized by low prevalence, a wide variety, and severe conditions, most closely related to gene mutations. With the rapid development of biotechnology and artificial intelligence, advanced therapies such as gene therapy and cell therapy have brought new breakthroughs in the research and development of drugs for rare diseases. This article systematically reviews 15 orphan drugs approved by the CBER under the FDA in 2024—2025 from two dimensions: indications and treatment modalities, with a focus on the application, challenges, and future trends of advanced therapies such as CRISPR-Cas9 gene editing technology and organoid models in the field of rare disease drug development. Additionally, the current status of China's rare disease drug research and development is discussed.
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| 科 Family | 属数 Number of genus | 种数 Number of species | 占总种数比例 Percentage of total species (%) | 属 Genus | 种数 Number of species | 占总种数比例 Percentage of total species (%) |
|---|---|---|---|---|---|---|
| 鹅膏菌科Amanitaceae | 2 | 11 | 5.26 | 鹅膏菌属 Amanita | 10 | 4.78 |
| 小菇科 Mycenaceae | 2 | 12 | 5.74 | 丝盖伞属 Inocybe | 5 | 2.39 |
| 多孔菌科 Polyporaceae | 8 | 14 | 6.70 | 蜡蘑属 Laccaria | 5 | 2.39 |
| 红菇科 Russulaceae | 3 | 23 | 11.00 | 小皮伞属 Marasmius | 6 | 2.87 |
| 小菇属 Mycena | 11 | 5.26 | ||||
| 光柄菇属 Pluteus | 5 | 2.39 | ||||
| 红菇属 Russula | 17 | 8.13 | ||||
| 栓菌属 Trametes | 5 | 2.39 |
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