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 Six most common mutations account for up to 26,000 patients in addressable markets (US,  Europe, Japan) with unique geographic mutational distribution; incidence rate of approximately  300 new patients per year Consistency in disease presentation and management across  mutations and key markets 

 In recent years, the field of psychiatric genetics has made strides in uncovering  common genetic threads that may underlie the coexistence of different psychiatric  disorders. Previous research, notably a large-scale effort by the Psychiatric Genomics  Consortium and other institutions in 2019, identified 136 regions in our genetic code  that are linked to eight major psychiatric disorders. Within these regions, they found 109  “hot spots” that were associated with more than one disorder, a phenomenon called  pleiotropy. 

 Currently, no drugs on the market can stop or reverse AD progression (Breijyeh and  Karaman, 2020). The acetylcholinesterase inhibitors donepezil, rivastigmine,  galantamine, huperzine A, and the N-methyl-D-aspartate receptor antagonist  memantine hydrochloride, which are the current clinical drugs for AD that can only  temporarily relieve the cognitive symptoms of patients (Breijyeh and Karaman, 2020).  Several new AD drugs have been terminated in clinical trials, including   

 As nanotechnology advances quickly, several nanomaterials can assist macromolecular  drugs and small-molecular drugs in penetrating through the BBB and successfully  delivering them to brain, which is important for developing new drugs and treating CNS  diseases (Wu et al., 2019; Jagaran and Singh, 2021; Zeng et al., 2021). More than 80  nanomedicine drugs have received worldwide regulatory approval, mainly antitumor  drugs, including doxorubicin liposomes, paclitaxel albumin nanoparticles, paclitaxel  polymer micelles, etc. (Ahmed and Qaisar, 2022). Nanomedicines can be loaded with  macromolecular drugs and small-molecular drugs to successfully cross the BBB through  masking, encapsulating, and embedding 

 Clinical treatments for AD currently focus primarily on symptom management and  supplemental brain nutrition. However, these methods offer palliative care without  addressing the underlying cause of the disease. The complexity of AD pathology and  the challenges associated with drug delivery across the BBB are the primary causes of  this limitation. Due to their excellent stability, biocompatibility, degradability, high safety,  flexible drug-loading methods, controllable drug release, and surface modifiability,  nanomedicine carriers have gained increasing attention. These characteristics make  them attractive candidates for targeted therapy in CNS diseases, 

 The number of patent applications for nanomedicine has significantly increased since  the 21st century due to the rapid development of nanotechnology, however, no  nanomedicines have yet been approved for treating CNS diseases. Clene  Nanomedicine, Inc., a clinical-stage biopharmaceutical company, has developed several  nanotechnology-based therapies for CNS diseases and initiated parallel phase II clinical  trials (NCT03843710, NCT03536559, NCT03815916, NCT04098406, NCT04626921,  and NCT05299658) with the lead asset CNM-Au8®. CNM-Au8® is a gold nanocrystal  suspension being researched as a disease-modifying treatment for patients with  amyotrophic lateral sclerosis, multiple sclerosis, and Parkinson’s disease (Kumar et al.,  2022; Vucic et al., 2023).