Recently, a patent titled "siRNA for Inhibiting APP Gene Expression, Its Conjugates, and Applications" by Beijing Youngen Biotechnology Co., Ltd., Ltd. has been officially authorized. This patent is a small nucleic acid drug patent for the treatment of Alzheimer's disease.
Currently, 10 million people worldwide are diagnosed with Alzheimer's disease every year, and there are approximately 50 million patients with Alzheimer's disease and other forms of dementia, bringing a heavy burden to society and families. The authorization of this related achievement undoubtedly brings a new dawn for the treatment of this disease.
Alzheimer's disease, commonly known as senile dementia, is the most common neurodegenerative disease. Its occurrence is associated with amyloid β plaques and neurofibrillary tangles. Patients may experience symptoms such as memory loss, or impairments in visual, language, executive, behavioral, and motor functions, which severely affect the quality of life. In-depth studies have found that the human APP (Amyloid Precursor Protein) gene plays a key role in this disease. This gene is located on chromosome 21q21.3, contains 20 exons, and encodes the amyloid precursor protein. The β-amyloid protein (Aβ) produced by the decomposition of APP membrane protein is an important pathological cause of Alzheimer's disease.
With aging, the expression of APP is induced, leading to the upregulation of β-secretase, thereby accelerating the accumulation of Aβ. Studies have found that transgenic mice overexpressing APP also undergo significant changes in vasoactive signals, resulting in neurovascular dysfunction. The reduction of APP will simultaneously reduce Aβ and all other toxic APP metabolites, which will alleviate the toxic environment related to Alzheimer's disease and slow down the progression of the disease. Therefore, reducing the formation of Aβ by targeting APP has become a focus of drug development.
However, current therapies targeting APP face many challenges. In immunotherapy, the effective delivery of drugs to the brain is a key issue, while other therapies have been terminated in clinical trials due to toxicity or side effects. Existing specific drugs for Alzheimer's disease, such as acetylcholinesterase inhibitors, can only alleviate the symptoms of mild to moderate Alzheimer's disease and have no significant therapeutic effect. Therefore, Alzheimer's disease still has unmet needs in many aspects, such as a large number of patients, low clinical treatment rate, and limitations of existing drugs.
Small nucleic acid drugs have a long half-life in the body, enabling administration once every six months or a year, which leads to good patient compliance. They are also expected to overcome the problems of difficult drug delivery and many side effects. The authorization of this related achievement provides new ideas and directions for solving the predicament of existing therapies, benefiting more patients.
