Biosplice’s neurodegeneration program is focused on a class of neurodegenerative conditions known as tauopathies. Tauopathies involve the abnormal aggregation of the protein tau, into neurotoxic, non-soluble aggregates and neurofibrillary tangles. Pathologic accumulation of tau is observed in neurodegenerative diseases such as Alzheimer’s disease, frontotemporal lobar degeneration with tau inclusions (FTLD-tau), and progressive supranuclear palsy, and Pick's disease, among others. Each of these diseases are characterized by high unmet need, with no approved treatments that slow or halt the progression of the disease.
Alzheimer’s disease, the most common cause of dementia and the most common tauopathy, is a chronic neurodegenerative disease affecting an estimated 5.8 million adults aged 65 and older in the U.S.1 and an estimated 50 million people worldwide.2
- Currently approved therapies help manage some symptoms but do not address the underlying causes or the progression of the disease, which is ultimately fatal.1
- With the world’s aging population, Alzheimer’s disease is quickly becoming a global epidemic, and a socioeconomic burden impacting families, social services, and healthcare delivery systems.1
Symptoms of Alzheimer’s disease generally appear in patients in their mid-60s, though symptoms may occur earlier in patients with familial forms of Alzheimer’s disease stemming from a genetic predisposition. The disease is initially characterized by progressive memory loss followed by slow progression to severe difficulty in accessing basic brain functions and subsequent mental disorders.
- Alzheimer’s Association. 2020 Alzheimer’s Disease Facts and Figures. Alzheimers Dement 2020;16(3):391+.
- Dementia statistics. www.alz.co.uk/research/statistics. Accessed 03/31/2020.